North American Cystic Fibrosis Conference
Minneapolis, Minnesota
October 15-17, 2009
1. Clancy, JP. “Clinical Trials of Lipid-Associated Aerosolized Amikacin: The Arikace™
Story” Pediatric Pulmonology Supp 32 (Summary S15.4), 2009.
2. Hutt, DM; Herman, DM; Rodrigues, AP; Noel, S; Gentzsch, M; Pilewski, JM; Riordan, JR;
Sorscher, EJ; Kelly, JW; Frizzell, RA; Manning, G; Gottesfeld, JM; Balch, WE. “Restoring
ΔF508-CFTR Trafficking and Activity with Epigenetic Modifiers” Pediatric Pulmonology
Supp 32 (Abst 8), 2009.
3. Protasevich, I; Yang, Z; Wang, C; Atwell, S; Zhao, X; Emtage, S; Wetmore, D; Hunt, J;
Brouillette, C. “Analysis of the Thermal Unfolding of CFTR Nucleotide Binding Domain 1
by Differential Scanning Calorimetry” Pediatric Pulmonology Supp 32 (Abst 41), 2009.
4. Zhang, S; Smith, N; Schuster, D; Sorscher, EJ; Rowe, SM; Woodworth, BA. “Activation of
Ciliary Beat Frequency in Primary Murine Nasal Epithelial Cultures by the CFTR
Potentiator, Quercetin” Pediatric Pulmonology Supp 32 (Abst 44), 2009.
5. DeLucas, LJ; Kappes, JC; Pruett, P; Ray, M; Dai, Q. “Large-Scale Expression and
Purification of CFTR” Pediatric Pulmonology Supp 32 (Abst 56), 2009.
6. Wang, C; Protasevich, I; Yang, Z; Atwell, S; Zhao, X; Emtage, S; Wetmore, D; Brouillette,
C; Hunt, J. “Integrated Biophysical Studies of HNBD1 Unfolding” Pediatric Pulmonology
Supp 32 (Abst 57), 2009.
7. Harris, WT; Kelly, DR; Hagood, JS. “Interstitial Remodeling: A Potential Link Between
TGF-Β1 and CF Lung Disease Histopathology” Pediatric Pulmonology Supp 32 (Abst 92),
2009.
8. Xu, X; Jackson, PL; Luy, G; Tanner, S; Hardison, MT; Blalock, JE; Gaggar, A.
“Acetylated PGP Induces Release of MMP-9 from Neutrophils via an ERK-Dependent
Pathway” Pediatric Pulmonology Supp 32 (Abst 107), 2009.
9. Liu, L; Bouma, BE; Rowe, SM; Tearney, GJ. “Video-Rate Ultrahigh-Resolution 3D Optical
Imaging of the Pulmonary Airways” Pediatric Pulmonology Supp 32 (Abst 120), 2009.
10. Virgin, FW; Wade, MB; St. John, JJ; Rowe, SM; Gaggar, A; Leon, KJ; Young, K;
Woodworth, BA “Aggressive Surgical & Postoperative Medical Management for Severe,
Recalcitrant Cystic Fibrosis Chronic Rhinosinusitis” Pediatric Pulmonology Supp 32 (Abst
156), 2009.
11. Plyler, ZE; Bernard, KF; Havasi, V; Keiles, SB; Hong, JS; Wen, H; Sorscher, EJ.
“Analysis of Synonymous CFTR Polymorphisms as Contributors to CBAVD” Pediatric
Pulmonology Supp 32 (Abst 188), 2009.
12. Chung, W; Rowe, SM; Keeton, AB; Shevin, R; Piazza, GA; Sorscher, EJ. “A Novel CellBased Imaging Assay for High Throughput Screens to Identify Small Molecule Correctors
of ΔF508-CFTR” Pediatric Pulmonology Supp 32 (Abst 192), 2009.
13. Chung, W; Fortenberry, JA; Kappes, JC; Accavitti-Loper, MA; DeLucas, LJ; Ray, MJ;
Alverson, P; Sorscher, EJ. “Development of Conformationally Sensitive Monoclonal
Antibodies Raised Against Full-Length CFTR” Pediatric Pulmonology Supp 32 (Abst
193), 2009.
14. Clancy, JP; Li, Y; Gentzsch, M. “Relationships Between Corrected ΔF508 and WT CFTR
Activity in CFBE41o- Cells in the Presence and Absence of Potentiators” Pediatric
Pulmonology Supp 32 (Abst 199), 2009.
15. Clancy, JP; Gabriel, S; Gentzsch, M; Donaldson, S; Hornick, D; Ahrens, R; Lymp, J;
Hocevar-Trnka, J. “Development of Multicenter GI Outcome Measures for CFTR
Modulator Clinical Trials” Pediatric Pulmonology Supp 32 (Abst 200), 2009.
16. Guo, J; Louie, R; Rodgers, JW; Miller, E; Sorscher, EJ; Hartman, JL. “Genome-Wide
Epistasis Analysis of a CFTR-ΔF508-Like Mutation in S. Cerevisiae” Pediatric
Pulmonology Supp 32 (Abst 208), 2009.
17. Solomon, GM; Konstan, MW; Wilschanski, M; Billings, J; Sermet, I; Accurso, FJ;
Vermeleun, F; Young, H; Reeves, G; Sabbatini, G; Mayer-Hamblett, N; Ashlock, M;
Clancy, JP; Rowe, SM. “Universal Protocol for Nasal Potential Difference Studies:
Results of an International Multi-Center Clinical Trial” Pediatric Pulmonology Supp 32
(Abst 215), 2009.
18. Rowe, SM; Backer, K; Tang, L; Woodworth, B; Mazur, M; Buckley-Lanier, J; Schwiebert,
E; Baasov, T; Bedwell, DM. “Enhanced Activity for Translational Readthrough and
Reduced Toxicity Demonstrated by the Synthetic Aminoglycoside NB54” Pediatric
Pulmonology Supp 32 (Abst 218), 2009.
19. Clancy, JP; Rowe, SM; Durie, P; Freedman, S; Dong, Q; Ordonez, C; Ashlock, M;
Accurso, FJ. “NPD Evaluation of Ion Transport in G551D CF Patients Treated with a
CFTR Potentiator” Pediatric Pulmonology Supp 32 (Abst 222), 2009.
20. Pyle, LC; Fan, L; Fortenberry, J; Tang, L; Backer, K; Mazur, M; Clancy, JP; Sorscher, EJ;
Rowe, SM. “Comprehensive Spectrum of Activity Analysis for ΔF508-CFTR Correctors”
Pediatric Pulmonology Supp 32 (Abst 229), 2009.
21. Goss, CH; Clancy, JP; Nick, JA; Billings, J; Rubenstein, RC; Young, KR; Sannuti, A;
Lechtzin, N; Nasr, SZ; Sawicki, GS; Quittner, AL; Lymp, JF; Hamblett, NM; Saiman, L;
Burns, JL; Ramsey, B; Gupta, R. “A Phase II Blinded and Placebo-Controlled Study of
Nebulized Liposomal Amikacin (Arikace™) in the Treatment of CF Patients with
Pseudomonas Aeruginosa Lung Infection” Pediatric Pulmonology Supp 32 (Abst 239),
2009.
22. Troxler, RB; Mobley, JA; Bowersock, G; Parrott, M; Roach, M; Sorscher, EJ. “Proteomic
Analysis of Salivary Fluid in Cystic Fibrosis” Pediatric Pulmonology Supp 32 (Abst 263),
2009.
23. Zhang, S; Schuster, D; Bedwell, D; Buckley-Lanier, J; Sorscher, EJ; Woodworth, BA.
“Marked Repression of CFTR MMA in the Transgenic CFTR^TM1KTH Mouse Model”
Pediatric Pulmonology Supp 32 (Abst 270), 2009.
24. Kong, M; Li, Y; Chen, F; Fan, L; Sullender, W; Clancy, JP. “Induction of MMP-9
Expression and Preferential Apical Surface Release Produced by RSV Infection of
Human Airway Cell Monolayers” Pediatric Pulmonology Supp 32 (Abst 309), 2009.
25. Troxler, RB; Hoover, WC; Britton, LJ; Gerwin, AM; Rowe, SM. “Eradication of Mucoid
Pseudomonas Aeruginosa in Patients with Cystic Fibrosis: A Retrospective Analysis”
Pediatric Pulmonology Supp 32 (Abst 330), 2009.
26. Gerwin, AM; Britton, LJ; Benner, K; Tofil, N; Guiterrez, H; Troxler, RB; Hoover, WC.
“Pseudomonas Aeruginosa Eradication in Cystic Fibrosis Patients: A Retrospective
Review” Pediatric Pulmonology Supp 32 (Abst 365), 2009.
27. Milla, C; Chmiel, JF; Accurso, FJ; McCoy, KS; Billings, JL; Atkinson, JJ; Clancy, JP; Liou,
TG; Acton, JD; Lynch, SV; Slusher, N; Burns, JL; Hamblett, NM; Harris, JK; Patel, R;
Tremblay, TM; Parli, TJ. “Anti-Inflammatory Effect of KB001, An Antipcrv Antibody
Fragment, in CF Patients Chronically Infected with Pseudomonas Aeruginosa” Pediatric
Pulmonology Supp 32 (Abst 369), 2009.
28. Britton, LJ; Hoover, W; Mims, C; Gutierrez, HH. “Reorganization of Annual Visits
Improves Clinic Efficiency and Satisfaction of Team, Patients and Families” Pediatric
Pulmonology Supp 32 (Abst 480), 2009.
29. Seals, NI; Britton, LJ; Gutierrez, H; Gunter, L; Wooldridge, N. ” Specialized Nutrition
Clinic Enhances Delivery of Nutrition Interventions and Improves Nutritional Status”
Pediatric Pulmonology Supp 32 (Abst 515), 2009.
30. Britton, LJ; Moreland, E; Wooldridge, N; Mims, C; Gutierrez, H; Rowe, SM; Hoover, W.
Development of a CFRD Clinic Improves Consistency of Care” Pediatric Pulmonology
Supp 32 (Abst 571), 2009.
31. Boren, KE; Gunter, LH; Gutierrez, H; Britton, LJ. “Maintaining Professional Boundaries”
Pediatric Pulmonology Supp 32 (Abst 608), 2009.
North American Cystic Fibrosis Conference
Baltimore, Maryland
October 21-23, 2010
1. Rowe, SM. “VX-770 from bench to the bedside and back again: Insights from CFTR
modulation in the clinic.” Pediatric Pulmonology Supp 33 (Summary S3.2), 2010.
2. Song W, Shipeng W, Matalon S. “Inhibition of lung fluid reabsorption and epithelial
sodium channels by respiratory syncytial virus.” Pediatric Pulmonology Supp 33
(Summary S6.1), 2010.
3. Rowe SM, Dransfield MT. “Lower airway potential difference measurements in COPD
subjects: Emerging evidence of acquired CFTR dysfunction.” Pediatric Pulmonology
Supp 33 (Summary S20.4), 2010.
4. Pyle LC, Balch W, Lukacs G, Braakman I, Guggino W, Thomas PJ, Penland C, Pollard H,
Brodsky J, Frizzell R, Sorscher EJ, Skach WR. “Developing a cellular roadmap for
correctors of CFTR misfolding.” Pediatric Pulmonology Supp 33 (Abst 7), 2010.
5. Sorscher EJ, Hong JS, Hutt DM, Chalfant M, Roth DM, Balch W, Noel S, Frizzell R,
Okiyoneda T, Veit G, Lukacs G, Eidelman O, Jozwik C, Caohuy H, Eudy Y, Pollard H,
Guggino B, Tessari MA, Fischer DF. “Screening of candidate adenovirus vectors
expressing shRNAs for functional recovery of ΔF508 CFTR.” Pediatric Pulmonology
Supp 33 (Abst 9), 2010.
6. Protasevich I, Yang Z, Wang C, Atwell S, Zhao X, Emtage S, Wetmore D, Hunt JF,
Brouillette C. “Analysis of the thermal unfolding of CFTR nucleotide binding domain 1
suggests a mechanism for the trafficking defect and temperature rescue of (F508del)
CFTR.” Pediatric Pulmonology Supp 33 (Abst 40), 2010.
7. Chung W, Ehrhardt A, Pyle LC, Wang W, Bear CE, Lukacs G, Kirk KL, Sorscher EJ. “A
biochemical assay for discovery of gating modulators based on intramolecular
interactions between nucleotide binding domain 1 and the first cytoplasmic loop of
CFTR.” Pediatric Pulmonology Supp 33 (Abst 47), 2010.
8. Li Y, Clancy JP. “Stability of C Band CFTR in T84 cell lysates with varying low
temperature storage conditions.” Pediatric Pulmonology Supp 33 (Abst 53), 2010.
9. Sabbatini GM, Donaldson SH, Dellom E, Bendahmane N, Quinney N, Gabriel S, Theresi
M, Starner TD, Hornick DB, Ernst S, Karp P, Fan L, Sugandha S, Clancy JP.
“Development of ex vivo GI outcome measures for studies of CFTR modulators.”
Pediatric Pulmonology Supp 33 (Abst 58), 2010.
10. Mitchell LH, Pyle LC, Bolger GB. “Activation of CFTR by the CAMP-specific
phosphodiesterase PDE4.” Pediatric Pulmonology Supp 33 (Abst 61), 2010.
11. McClure M, DeLucas LJ, Wilson L, Ray M, Hong JS, Kappes JC, Sorscher EJ, Barnes S.
“Post-translational modifications of CFTR with relevance to gating and processing.”
Pediatric Pulmonology Supp 33 (Abst 62), 2010.
12. Harris WT, Grenett HE, Macewan M, Clancy JP. “TGFβ1 activation in cystic fibrosis cell
culture model systems.” Pediatric Pulmonology Supp 33 (Abst 85), 2010.
13. Solomon GM, Frederick C, Sabbatini G, Gaggar A, Harris T, Woodworth BA, Steele C,
Rowe SM. “IP-10 is a potential biomarker of acute pulmonary exacerbations of cystic
fibrosis in nasal lavage.” Pediatric Pulmonology Supp 33 (Abst 105), 2010.
14. Tuggle KL, Asmellash S, Dickinson DA, Mobley JA, Fanucchi MV. “Early life exposure to
ozone alters airway epithelial responses in wild-type and cystic fibrosis transmembrane
receptor (CFTR) knockout mice.” Pediatric Pulmonology Supp 33 (Abst 133), 2010.
15. Zhang S, Skinner D, Hicks B, Bevensee M, Sorscher EJ, Woodworth BA. “Sinupret®
stimulates ciliary beat frequency and transepithelial chloride transport through CFTR
dependent and independent mechanisms.” Pediatric Pulmonology Supp 33 (Abst 137),
2010.
16. Skinner D, Zhang S, Azbell C, Fortenberry J, Sorscher EJ. “Hesperidin stimulates CFTRmediated chloride secretion and ciliary beat frequency in sinonasal epithelium.” Pediatric
Pulmonology Supp 33 (Abst 138), 2010.
17. Guo J, Louie R, Rodgers JW, Miller E, Sorscher EJ, Hartman JL. “YOR1-ΔF as a CFTRΔF508-like mutation to identify modulators in S. cerevisiae.” Pediatric Pulmonology Supp
33 (Abst 201), 2010.
18. Havasi V, Buckley-Lanier JA, Childress Roper V, Fortenberry JA, Bedwell DM, Sorscher
EJ, Yoder BK. “Development of a CFTR deficient ciliopathic mouse model.” Pediatric
Pulmonology Supp 33 (Abst 215), 2010.
19. Liu L, Shastry S, Sloane PA, Mazur M, Parker SB, Bouma BE, Tearney GJ, Rowe SM.
“Modulation of ion transport and mucus clearance in cells and tissues: New insights from
video-rate reflectance tomography imaging.” Pediatric Pulmonology Supp 33 (Abst 222),
2010.
20. Clancy JP, Rowe SM, Accurso FJ, Ballmann M, Boyle MP, DeBoeck C, Konstan MW,
Spencer-Green G. “A Phase II, randomized, placebo-controlled, clinical trial of four doses
of VX-809 in CF patients homozygous for the F508del CFTR mutation.” Pediatric
Pulmonology Supp 33 (Abst 224), 2010.
21. Clancy JP, Rowe SM, Durie PR, Konstan MW, Dunitz J, Hornick DB, Sagel SD, Boyle
MP, Uluer AZ, Moss RB, Freedman S, Dong Q, Zha J, Stone A, Olson E, Ordonez C,
Campbell P, Ashlock M, Accurso FJ. “Comparison of NPD parameters in a phase IIa
study to optimize detection of CFTR modulator bioactivity in clinical trials.” Pediatric
Pulmonology Supp 33 (Abst 231), 2010.
22. Lui B, Hathorne H, Hill A, Hovick G, Cohen M, Beamer J, Solomon MG, Clancy JP, Rowe
SM. “Normative values and receiver operating characteristics of NPD for diagnostic
measurements.” Pediatric Pulmonology Supp 33 (Abst 247), 2010.
23. Clancy JP, Minic P, Dupont L, Goss CH, Quittner AL, Lymp JF, Burns JL, Govan JR,
Ramsey B, Gupta R. “Full analyses of data from two phase II blinded and placebocontrolled studies of nebulized liposomal amikacin for inhalation (arikaceTM) in the
treatment of CF patients with Pseudomonas aeruginosa lung infection.” Pediatric
Pulmonology Supp 33 (Abst 227), 2010.
24. Sloane PA, Tang L, Mazur M, Buckley-Lanier J, Bedwell D, Rowe SM. “Translational
readthrough of premature stop codons combined with CFTR potentiation: Potential for
combination CFTR therapy.” Pediatric Pulmonology Supp 33 (Abst 264), 2010.
25. Kerem E, Wilschanski M, Melotti P, Sermet-Gaudelus I, DeBoeck K, Rowe SM, Konstan
MW, Accurso F, Pimentel S, Constantine S, Miller N, Barth J, Miller L, Ajayi T. “Phase 3
study of Ataluren (PTC124®) in nonsense mutation cystic fibrosis (NMCF): Demographic
and other baseline data.” Pediatric Pulmonology Supp 33 (Abst 266), 2010.
26. Rowe SM, van Goor F, Clancy JP, Durie PR, Konstan MW, Dunitz J, Hornick DB, Sagel
SD, Boyle MP, Uluer AZ, Moss RB, Ramsey B, Freedman S, Dong Q, Zha J, Stone A,
Olson E, Ordonez C, Campbell P, Ashlock M, Accurso FJ. “Corresponding effects of VX770 on NPD in vivo and human bronchial epithelial (HBE) cells in vitro.” Pediatric
Pulmonology Supp 33 (Abst 281), 2010.
27. Merriss M, Thompson P, Britton LJ, Troxler RB, Hoover W. “Burkholderia cepacia
eradication in cystic fibrosis patients: A retrospective review.” Pediatric Pulmonology
Supp 33 (Abst 338), 2010.
28. Maeng P, Li Y, Hong JS, Gaggar A, Clancy JP, Kong M. “RSV infection stimulates MMP9 expression and release from human airway monolayers, and replication is abrogated by
MMP-9 inhibition.” Pediatric Pulmonology Supp 33 (Abst 362), 2010.
29. Coats M, Nahm MH, Briles DE, Crain MJ. “Mucoid pneumococci are unusually common
in the cystic fibrosis lung.” Pediatric Pulmonology Supp 33 (Abst 372), 2010.
30. Zhang S, Skinner D, Sorscher EJ, Woodworth BA. “LPS and Pseudomonas aeruginosa
filtrate reduce calcium activated chloride channel transport in primary sinonasal epithelial
cultures.” Pediatric Pulmonology Supp 33 (Abst 382), 2010.
31. Sabbatini GM, Britton LJ, Hathorne HY, Reeves GA, Lucious TD. “Integration of clinical
and research terms improve patient enrollment in clinical trials.” Pediatric Pulmonology
Supp 33 (Abst 501), 2010.
32. Britton LJ, Boren KE, Hoover W, Rodgers TF, Gutierrez HH. “Assessment of team
satisfaction, meeting quality, and team cohesiveness.” Pediatric Pulmonology Supp 33
(Abst 517), 2010.
33. Dharmalingam P, Kurundkar A, Harris WT, Clancy JP, Sorscher EJ, Maheshwari A.
“Intestinal mucosal inflammation in cystic fibrosis.” Pediatric Pulmonology Supp 33 (Abst
572), 2010.
34. Wooldridge N, Anderson V, Gutierrez H, Gardner J, Britton LJ. “The relationship between
suboptimal serum 25-hydroxyvitamin D levels and cystic fibrosis related diabetes.”
Pediatric Pulmonology Supp 33 (Abst 587), 2010.
35. Christian BJ. “Children’s perceptions of growing up with cystic fibrosis: Discovering the
chronic illness trajectory.” Pediatric Pulmonology Supp 33 (Abst 614), 2010.
36. Britton LJ, Mims C, Brown J, Troxler B. “Nursing annual visit questionnaire to assess
patient and family knowledge of CF.” Pediatric Pulmonology Supp 33 (Abst 636), 2010.
37. Self S, Gutierrez H, Britton LJ, Troxler B. “Evaluation of different education tracks for
parents of babies diagnosed with CF through newborn screening.” Pediatric Pulmonology
Supp 33 (Abst 650), 2010.
North American Cystic Fibrosis Conference
Anaheim, CA
November 3-5, 2011
1. Kirk, KL. “ATP-free CFTR channel gating: Constitutive mutants, R domain regulation and
CF relevance.” Pediatric Pulmonology Supp 34 (Summary S4.4), 2011.
2. Rowe, SM. “Measuring CFTR activity in clinical trials: in vivo assays.” Pediatric
Pulmonology Supp 34 (Summary S14.2), 2011.
3. Rowe, SM; Boyle MP. “Combination of correctors and potentiators for F508del CFTR.”
Pediatric Pulmonology Supp 34 (Summary S15.3), 2011.
4. Hudson, R; Chong, P; Dawson, JE; Mense, M; Thomas PJ; Brouillette, C; Forman-Kay,
JD “Common conformational changes within NBD1 elicited by CF modulators, an ICL4
peptide and suppressor mutations.” Pediatric Pulmonology Supp 34 (Abst 7), 2011.
5. Chung, W; Fortenberry, JA; Kappes, JC; DeLucas, LJ; Marjorie, RJ; Alverson, P;
Sorscher, EJ; Accavitti-Loper, M. “Towards development of conformationally sensitive
monoclonal antibodies against CFTR.” Pediatric Pulmonology Supp 34 (Abst 26), 2011.
6. Chung, W; Rowe, SM; Sorscher, EJ; Piazza, GA; Keeton, AB. “High content cell-based
imaging to identify small molecule correctors of F508del-CFTR.” Pediatric Pulmonology
Supp 34 (Abst 27), 2011.
7. Wang, C; Protassevitch, I; An, J; Kota, P; Dokholyan, N; Brouillette, C; Hunt, J.
“Engineering a cys-less human NBD1 for use in visible fluorescence assays of domain
stability and intermolecular associations.” Pediatric Pulmonology Supp 34 (Abst 46),
2011.
8. McClure, M; Wilson, L; DeLucas, LJ; Ray, M; Rowe, SM; Wu, X; Dai, Q; Hong, J;
Kappes, JC; Sorscher, EJ; Barnes, S. “Identification of CFTR post-translational
modification by mass spectrometry.” Pediatric Pulmonology Supp 34 (Abst 64), 2011.
9. McClure, M; Wilson, L; DeLucas, LJ; Ray, M; Rowe, SM; Wu, X; Dai, Q; Hong, JS;
Kappes, JC; Barnes, S; Sorscher, EJ. “S-palmitoylation is a regulator of CFTR
processing.” Pediatric Pulmonology Supp 34 (Abst 65), 2011.
10. Liu, L; Shastry, S; Sloane, PA; Mazur, M; Parker, S; Grizzle, W; Bouma, BE; Rowe, SM;
Tearney, GJ. “Visualizing respiratory ciliary motion and mechanosensitivity of ciliated
cells using 1-mm resolution optical coherence tomography (MOCT).” Pediatric
Pulmonology Supp 34 (Abst 71), 2011.
11. Maeng, P; Kong, M; Hong, J; Sorscher, E; VanDyke, R; Clancy, J. “RSV infection rapidly
disrupts airway cell monolayer integrity in cells expressing F508del CFTR, but not in
monolayers expressing wt CFTR.” Pediatric Pulmonology Supp 34 (Abst 78), 2011.
12. Sabbatini, GM; Xu, X; Gaggar, A. “C-reactive protein as a potential biomarker predictive
for inpatient exacerbation in cystic fibrosis.” Pediatric Pulmonology Supp 34 (Abst 81),
2011.
13. Sanders, Y; Harris, T; Li, Y; Clancy, JP. “TGF-beta down-regulates CFTR and TMEM16A
expression and function in polarized T84 cells.” Pediatric Pulmonology Supp 34 (Abst
93), 2011.
14. Skinner, D; Alexander, NS; Hatch, N; Zhang, S; Fortenberry, J; Sorscher, EJ;
Woodworth, BA. “Resveratrol has salutary effects on mucociliary transport and
inflammation in sinonasal epithelium.” Pediatric Pulmonology Supp 34 (Abst 108), 2011.
15. Zhang, S; Blount, A; Chestnut, M; Hixon, B; Skinner, D; Sorscher, EJ; Woodworth, BA.
“Transepithelial ion transport through CFTR and TMEM16A is suppressed in hypoxic
sinonasal epithelium.” Pediatric Pulmonology Supp 34 (Abst 109), 2011.
16. Woodworth, BA; Zhang, S; Skinner, D; Sorscher, EJ; Rowe, SM. “Comparison of CFTR
and ciliary beat frequency activation by the CFTR modulators VX-770, VRT532, and
UCCF-152 in primary sinonasal epithelial cultures.” Pediatric Pulmonology Supp 34 (Abst
114), 2011.
17. Hong, J; Guo, J; Louie, R; Wen, H; Sorscher, EJ; Miller, E; Hartman, JL. “A probiogenesis complex for F508del-CFTR discovered with a yeast model and validated in
human cells.” Pediatric Pulmonology Supp 34 (Abst 178), 2011.
18. Stalvey, MS; Cass, S; Gaur, T; Flotte, TR; Lian, JB; Stein, GS; Clines, KL; Chung, WJ;
Clines, GA. Cystic fibrosis bone disease: Mechanisms of reduced proliferation and
delayed osteogenic differentiation of CFTR deficient mesenchymal stromal cells.
Pediatric Pulmonology Supp 34 (Abst 180), 2011.
19. Harris, WT; Nicola, T; Macewen, M; Li, Y; Ambalavanan, N; Clancy, JP. “TGF-Β
production and myofibroblast differentiation in cystic fibrosis.” Pediatric Pulmonology
Supp 34 (Abst 184), 2011.
20. Guo, J; Louie, R; Rodgers, JW; White, R; Hong, J; Sorscher, EJ; Bryan, JA; Miller, EA;
Hartman, JL. “A conserved biogenesis network for yeast YOR1-ΔF and CFTR-ΔF.”
Pediatric Pulmonology Supp 34 (Abst 185), 2011.
21. Havasi, V; Buckley-Lanier, J; Childress-Roper, V; Fortenberry, JA; Schoeb, TR; Bedwell,
DM; Yoder, BK; Sorscher, EJ. “Developing a CFTR deficient PKD mouse model.”
Pediatric Pulmonology Supp 34 (Abst 193), 2011.
22. Clancy, JP; Sun, H; Donaldson, SH; Gabriel, S; Starner, TD; Ahrens, R; Hornick, DB; Liu,
B; Rowe, SM. “CF and non-CF intestinal biopsy currents defining CFTR function through
ROC curve analyses and ex vivo modulation.” Pediatric Pulmonology Supp 34 (Abst
199), 2011.
23. Clancy, JP; Rowe, SM; Liu, B; Hathorne, H; Dong, Q; Wisseh, S; Ordonez, C. “Variability
of nasal potential difference measurements in clinical testing of CFTR modulators.”
Pediatric Pulmonology Supp 34 (Abst 202), 2011.
24. Konstan, MW; Accurso, FJ; DeBoeck, K; Kerem, E; Rowe, SM; Sermet-Gaudelus, I;
Wilschanski, M; Miller, N; Elfring, GL; Barth, J; Ajayi, T. “Pretreatment data from phase 3
study of ataluren document significant disease burden in a subpopulation of patients with
nonsense mutation cystic fibrosis.” Pediatric Pulmonology Supp 34 (Abst 232), 2011.
25. Sabbatini, GM; Frederick, C; Konstan, MW; Rosenbluth, D; Liu, B; Bucur, C; Burks, P; Li,
Y; Lymp, JF; Clancy, JP; Rowe, SM. “Nasal potential difference studies utilizing the
CFTR modulator quercetin.” Pediatric Pulmonology Supp 34 (Abst 244), 2011.
26. Sloane, PA; Shastry, S; Wilhelm, A; Courville, C; Tang, l; Mehta, N; Backer, K; Levin, E;
Mazur, M; Van Goor, F; Byan-Parker, S; Grizzle, W; Sorscher, EJ; Dransfield, MT; Rowe,
SM. Pharmacologic rescue of acquired CFTR dysfunction in chronic obstructive
pulmonary disease. Pediatric Pulmonology Supp 34 (Abst 245), 2011.
27. Ballew, B; Troxler, B; Britton, LJ; Hoover, W; Searcey, H; Cates, M. Clinical outcomes in
cystic fibrosis patients prescribed thrice weekly azithromycin without chronic infection
with Pseudomonas aeruginosa. Pediatric Pulmonology Supp 34 (Abst 348), 2011.
28. Hoover, W; Mims, C; Gamble, S. Sensitivity and specificity of infant pulmonary function
testing in sick and well CF infants identified by newborn screening. Pediatric
Pulmonology Supp 34 (Abst 374), 2011.
29. Gutierrez, H; Self, S; Gamble, S. Statistical process control (SPC)—validated home
spirometry for CF lung disease. Pediatric Pulmonology Supp 34 (Abst 380), 2011.
30. Hathorne, H; Britton, LJ; Reeves, GA; Sabbatini, GM; Tarn, VE. Initiation of quality
improvement within the clinical research team. Pediatric Pulmonology Supp 34 (Abst
441), 2011.
31. Grant, AP; Britton, LJ; Wooldridge, N; Gutierrez, H. Room service for the patient with
cystic fibrosis: Patient and staff satisfaction survey. Pediatric Pulmonology Supp 34 (Abst
469), 2011.
32. Gutierrez, H; Self, S; Mims, C. Use of checklists to assist patients with early recognition
of acute pulmonary exacerbation in cystic fibrosis. Pediatric Pulmonology Supp 34 (Abst
473), 2011.
33. Bailey, J; Wooldridge, N; Redden, D; Britton, LJ; Hoover, W; Gutierrez, H. Predictors of
vitamin D insufficiency in pediatric patients with cystic fibrosis. Pediatric Pulmonology
Supp 34 (Abst 509), 2011.
34. Stalvey, MS; Cass, S; Mueller, C; Flotte, TR. “CFTR knockout mice reveal normal
glucagon response to hypoglycemia.” Pediatric Pulmonology Supp 34 (Abst 524), 2011.
North American Cystic Fibrosis Conference
Orlando, FL
October 11-13, 2012
1.
Rowe, S; Tearney, G, J. “Functional anatomic imaging using one micron resolution
optical coherence tomography.: Pediatric Pulmonology Supp 35 (Summary S16.3), 2012.
2. Konstan, M; Accurso, F; De Boeck, K; Kerem, E; Rowe, S; Sermet-Gaudelus, I;
Wilschanski, M; Barth, J; Elfring, G; Peltz, S; Ajayi, T. “Targeting class 1 mutations:
Update on ataluren as a promising treatment for nonsense mutation cystic fibrosis.”
Pediatric Pulmonology Supp 35 (Summary S1.1), 2012.
3.
Thomas, PJ; Schmidt, A; Mendoza, JL; Millen, L; Richardson, J; Fuller, M; Patrick, A;
Vetter, A; Karamysheva, Z; Karamyshev, AL. “Mechanism of action and molecular
pathology: Matching drugs and mutants.” Pediatric Pulmonology Supp 35 (Summary
S8.2), 2012.
4. Chung, WJ; Goeckeler-Fried, J; Brodsky, J; Havasi, V; Rowe, SM; Piazza, GA; Keeton,
AB; Sorscher, EJ. “Availability of F508del CFTR for small molecule correction.” Pediatric
Pulmonology Supp 35 (Abst 4), 2012.
5. Chung, WJ; Fortenberry, JA; Lukacs, G; Roldan, HA; Kappes, JC; Brouillette, C;
DeLucas, LJ; Ray, MJ; Sorscher, EJ; Accavitti-Loper, MA. “Developing antibodies
capable of monitorind conformation and maturation of CFTR.” Pediatric Pulmonology
Supp 35 (Abst 5), 2012.
6. Aller, S; Tang, LP; Rajpur, J; Sloane, P; Sorscher, EJ; Rowe, SM. “Identification of
efficacious F508del CFTR correctors using a novel homology model.” Pediatric
Pulmonology Supp 35 (Abst 12), 2012.
7. Goeckeler-Fried, J; Chiang, A; Chung, W. J; Havasi, V; Weissman, AJ; Lewis, T;
Brodsky, JL. “Synergistic correction of ΔF508-CFTR when an inhibitor of protein
ubiquitination is combined with a small molecule corrector.” Pediatric Pulmonology Supp
35 (Abst 33), 2012.
8. Wang, C; Kota, P; An, J; Protassevitch, I; Dokholyan, NV; Brouillette, C; Hunt, JF. “A
robust and efficient visible fluorescence assay for human NBD1 folding and stability
designed for mechanistic studies and high-throughput screening for corrector
compounds.” Pediatric Pulmonology Supp 35 (Abst 50), 2012.
9. Hudson, R; Chong, PA; Protasevich, II; Dawson, J; Vernon, R; Noy, E; Bihler, H; An, JL;
Kalid, O; Sela-Culang, I; Mense, M; Senderowitz, H; Brouillette, C; Forman-Kay, J.
“Direct binding of CFTR modulators to human CFTR NBD1.” Pediatric Pulmonology Supp
35 (Abst 59), 2012.
10. Yang, Z; Wang, C; Zhou, Q; An, J; Hunt, JF; Brouillette, C. ”The importance of the NBD
domains in the selection of detergents for CFTR purification.” Pediatric Pulmonology
Supp 35 (Abst 67), 2012.
11. Rooj, AK; Cormet-Boyaka, E; Clark, EB; Qadri, YJ; Sorscher, EJ; Fuller, CM; Berdiev,
BK. “Association of CFTR with ENaC subunits carrying Liddle’s Syndrome mutations.”
Pediatric Pulmonology Supp 35 (Abst 68), 2012.
12. McClure, M; Wilson, L; Ray, M; DeLucas, LJ; Rowe, S; Wu, X; Dai, Q; Hong, J; Sorscher,
EJ; Kappes, JC; Barnes, S. “Post-translational modifications of CFTR glycoforms
identified by high resolution, high mass accuracy mass spectrometry.” Pediatric
Pulmonology Supp 35 (Abst 70), 2012.
13. McClure, M; Wilson, L; Ray, M; DeLucas, LJ; Rowe, S; Wu, X; Dai, Q; Hong, JS; Kappes,
JC; Barnes, S; Sorscher, EJ. “S-palmitoylation mediates trafficking of CFTR to the cell
surface.” Pediatric Pulmonology Supp 35 (Abst 71), 2012.
14. Zhang, S; Skinner, D; Chaaban, M; Sorscher, E; Woodworth, BA. “CFTR activation by
the solvent ethanol: Implications for CF drug testing and delivery.” Pediatric Pulmonology
Supp 35 (Abst 75), 2012.
15. DeLucas, LJ; Kappes, JC; Dai, Q; Ray, M; Brackin, W; McCombs, D; Johnson, D; Ding,
H; Hildebrandt, E; Purna, B; Riordan, JR; Urbatsch, IL. “Progress toward elucidating the
3D structure of full-length CFTR.” Pediatric Pulmonology Supp 35 (Abst 78), 2012.
16. Raju, SV; Jackson, PL; McNicholas, CM; Sloane, PA; Sabbatini, GM; Tang, LP; Mazur,
M; Liu, B; Clancy, JP; Bowen, L; Accurso, F; Blalock, JE; Dransfield, M; Rowe, SM.
“Defects in CFTR activity can be acquired and transmitted by a systemic pathway.”
Pediatric Pulmonology Supp 35 (Abst 81), 2012.
17. Harris, WT; Kelly, D; Hagood, J; Ambalavanan, N; Sorscher, E. “Myofibroblast
proliferation and fibrosis in CF lung disease.” Pediatric Pulmonology Supp 35 (Abst 106),
2012.
18. Szul, T; Gaggar, A; Xu, X. “Expression of prolyl endopeptidase in bronchial epithelial
cells: Impact of F508del CFTR.” Pediatric Pulmonology Supp 35 (Abst 110), 2012.
19. McNicholas, CM; Gaggar, A. “Neutrophil elastase modulates CFTR current in bronchial
epithelial cells.” Pediatric Pulmonology Supp 35 (Abst 125), 2012.
20. Jackson, PL; Xu, X; Davis, V; Jones, C; Okafor, S; Noerager, B. “Modulation of small
matrikines by reactive aldehydes in CF.” Pediatric Pulmonology Supp 35 (Abst 150),
2012.
21. Tuggle, KL; Gamber, K; Cui, X; Ji, D; Chambers, A; Bedwell, DM; Rowe, SM; Sorscher,
EJ; Fanucchi, MV. “Generation of a CFTR-knockout rat model for cystic fibrosis.”
Pediatric Pulmonology Supp 35 (Abst 162), 2012.
22. Guo, J; Sorscher, EJ; Hartman, JL. “Advancing a yeast phenomics model for gene
interaction network analysis of F508del-CFTR biogenesis.” Pediatric Pulmonology Supp
35 (Abst 184), 2012.
23. Skinner, D; Zhang, S; Chaaban, M; Sorscher, E; Woodworth, BA. “Primary rabbit nasal
septal epithelial cultures for studies of cystic fibrosis sinus disease.” Pediatric
Pulmonology Supp 35 (Abst 188), 2012.
24. Rowe, S; Sermet-Gaudelus, I; Konstan, M; Kerem, E; Wilschanski, M; DeBoeck, K;
Accurso, F; Miller, NL; Elfring, GL; Barth, J; Ajayi, T. “Results of the phase 3 study of
ataluren in nonsense mutation cystic fibrosis (NMCF).” Pediatric Pulmonology Supp 35
(Abst 193), 2012.
25. Rowe, SM; Tang, LP; Xue, X; Biswas, S; Du, M; Belakhov, V; Kandasamy, J; Chen, F;
Schacht, J; Baasov, T; Bedwell, D. “The synthetic aminoglycoside NB124 suppresses
CFTR premature termination codons more effectively than gentamicin and prior synthetic
derivatives.” Pediatric Pulmonology Supp 35 (Abst 194), 2012.
26. Xu, X; Sabbatini, GM; Hathorne, H; Clancy, JP; Gaggar, A. “A randomized double blinded
placebo control trial of doxycycline as an adjunctive anti-inflammatory agent during CF
exacerbation (DOXY).” Pediatric Pulmonology Supp 35 (Abst 209), 2012.
27. Clancy, JP; Donaldson, S; Gabriel, S; Hornick, D; Starner, TD; Rowe, S; VanDyke, R;
Fenchel, M; Sun, H. “Centralized intestinal current measurements (ICM): CFTR activity in
fresh and cold-stored rectal biopsies, and comparison of forceps vs. suction biopsy.”
Pediatric Pulmonology Supp 35 (Abst 210), 2012.
28. Kerem, E; Wilschanski, M; Accurso, F; DeBoeck, K; Konstan, M; Rowe, S; SermetGaudelus, I; Miller, N; Elfring, GL; Barth, J; Ajayi, T. “Natural history of cystic fibrosis in
patients with nonsense-mutation-mediated disease.” Pediatric Pulmonology Supp 35
(Abst 252), 2012.
29. Liu, L; Wilsterman, E; Shastry, S; Houser, G; Smith, A; Boylan, N; Hanes, J; Tearney, G;
Rowe, SM. “Measuring mucus viscosity using micro-optical coherence tomography
developed microrheology techniques.” Pediatric Pulmonology Supp 35 (Abst 255), 2012.
30. Kong, M; Peng, N; Clancy, JP; Blalock, E; Gaggar, A; Sorscher, EJ. “”RSV stimulates
MMP-9 release from CF epithelial cells and increases MMP activity in human lungs.”
Pediatric Pulmonology Supp 35 (Abst 263), 2012.
31. Dennis, E; Coats, M; Briles, D; Crain, M. “The effects of CFTR and capsular phenotype
on susceptibility and innate immune responses in a mouse model of pneumococcal lung
disease.” Pediatric Pulmonology Supp 35 (Abst 303), 2012.
32. Searcy, H; Phillips, S; Benner, K; Britton, LJ; Harris, WT; Hoover, W. “Eradication of
Pseudomonas aeruginosa in pediatric patients with cystic fibrosis: A comparison of
protocols.” Pediatric Pulmonology Supp 35 (Abst 346), 2012.
33. Harris, WT; Boyd, JT; VanDyke, R; Fenchel, M; Brody, A; McPhail, GL; Clancy, JP. “CFrelated constrictive bronchiolitis in adolescents with refractory pulmonary decline.”
Pediatric Pulmonology Supp 35 (Abst 372), 2012.
34. Searcy, H; Britton, LJ; Askenazi, D; Scott, A; Lyrene, R; Gutierrez, H. “Prevention of
acute kidney injury in hospitalized children with cystic fibrosis.” Pediatric Pulmonology
Supp 35 (Abst 430), 2012.
35. Britton, LJ; Mims, C; Harris, WT; Brown, J. “Predictors of parental knowledge and patient
outcomes in patients diagnosed by newborn screening.” Pediatric Pulmonology Supp 35
(Abst 436), 2012.
36. Rodgers, TF; Britton, LJ; Woodridge, N; Stalvey, MS; Gutierrez, H; Hoover, W.
“Mesosystems approach to cystic fibrosis related diabetes (CFRD) management.”
Pediatric Pulmonology Supp 35 (Abst 477), 2012.
37. Britton, LJ; Gutierrez, H; Hardy, J. “Patient acceptance and satisfaction with complete
segregation while hospitalized.” Pediatric Pulmonology Supp 35 (Abst 490), 2012.
38. Hoover, W; Phillips, C; Benner, K; Britton, LJ; Harris, WT; Searcy, H. “Effect of proton
pump inhibitors on absorption and serum levels of vitamin D in cystic fibrosis patients.”
Pediatric Pulmonology Supp 35 (Abst 533), 2012.
39. Kines, K; Wooldridge, N; Griffin, R; Britton, LJ; Hoover, W; Gutierrez, H. “Nutritional
status and disease severity in pre-adolescence and adolescent patients with cystic
fibrosis.” Pediatric Pulmonology Supp 35 (Abst 535), 2012.
40. Stalvey, MS; Clines, KL; Chung, WJ; Clines, GA. “Cystic fibrosis bone disease:
Interactions between osteoblasts and osteoclasts may be the key to increased bone
loss.” Pediatric Pulmonology Supp 35 (Abst 541), 2012.
41. Grossoehme, DH; VanDyke, R; Dimitriou, S; Britton, LJ; McPhail, GL; Seid, M. “Airway
clearance adherence: Influence of parental spirituality and depression.” Pediatric
Pulmonology Supp 35 (Abst 576), 2012.
42. Loosen, H; Vela, K; Klintworth, T; Lew, J; Lowman, J. “On the Go with ECMO! Preparing
Clinical Patients for Transplant with Exercise & Ambulation” (Care) Pediatric
Pulmonology Supp 35. 2012.
North American Cystic Fibrosis Conference
Salt Lake City, Utah
October 17-19, 2013
1. Rowe, SM. “The G551D observational trial (goal): New insights into CFTR modulation
from the clinic.” Pediatric Pulmonology Supp 36 (Summary S10.4), 2013.
2. Seakwoo, L; McClure, M; Wilson, L; Sorscher, E; Zeitlin, P. “Interference with
ubiquitination of lysines in the NTAIL, R, TM10, ICL4, or NBD2 domains modifies stability
of wt CFTR.” Pediatric Pulmonology Supp 36 (Abst 3), 2013.
3. Veit, G; Okiyoneda, T; Oliver, K; Perdomo, D; Sorscher, EJ; Hartman IV, JL; Lukacs, GL.
“Translational relevance of the yeast YOR1-ΔF670 model for ΔF508-CFTR biogenesis.”
Pediatric Pulmonology Supp 36 (Abst 14), 2013.
4. Roessler, BC; Wei, S; Hartman, JL; Kirk, KL. “Computational and functional experiments
reveal similarities between the CFTR channel and ABC export pumps.” Pediatric
Pulmonology Supp 36 (Abst 28), 2013.
5. McClure, M; Hong, JS; Wen, H; Sorscher, EJ. “S-palmitoylation regulates stability of core
glycosylated wild-type and F508del CFTR in a post-ER compartment.” Pediatric
Pulmonology Supp 36 (Abst 42), 2013.
6. Wang, W; Kirk, KL. “Interaction between E267 and K1060 in the CFTR coupling helices
promotes channel opening.” Pediatric Pulmonology Supp 36 (Abst 47), 2013.
7. Lambert, JA; Raju, SV; Farris, R; Tang, LP; Li, Y; Courville, C; Coricor, G; Smoot, L;
Mazur, M; Dransfield, M; Bolger, G; Rowe, SM. “CFTR activation by roflumilast
contributes to therapeutic benefit in chronic bronchitis.” Pediatric Pulmonology Supp 36
(Abst 54), 2013.
8. Hudson, R; Dawson, JE; Chong, PA; Brouillette, C; Forman-Kay, J. “The effect of RI, RE,
and ICL4 interactions on the binding of CFFT-001 and VX-809 to isolated NBD1.”
Pediatric Pulmonology Supp 36 (Abst 58), 2013.
9. Chung, WJ; Goeckeler-Fried, J; Brodsky, J; Havasi, V; Rasmussen, L; White, EL;
Sorscher, EJ. “High throughput screening for compounds that augment the pool of
F508del CFTR suitable for pharmacologic correction.” Pediatric Pulmonology Supp 36
(Abst 62), 2013.
10. Chong, PA; Hudson, R; Vernon, RM; Forman-Kay, J. “F508del impairment of NBD
dimerization.” Pediatric Pulmonology Supp 36 (Abst 63), 2013.
11. Wang, C; Kota, P; An, J; Protassevitch, I; Dokholyan, NV; Brouillette, C; Hunt, JF.
“Chimeric transmembrane protein for characterization of the NBD1-TMD interface in
human CFTR.” Pediatric Pulmonology Supp 36 (Abst 72), 2013.
12. Hildebrandt, E; Kappes, JC; DeLucas, LJ; Cant, N; Ford, R; Zhang, Q; Urbatsch, IL.
“Evaluation of detergents for CFTR purification, concentration, and reconstitution of
ATPASE activity.” Pediatric Pulmonology Supp 36 (Abst 73), 2013.
13. Mutyam, V; Xue, X; Jackson, L; Hong, J; Biswas, S; Bridges, R; Baasov, T; Belakhov, V;
Bedwell, D; Rowe, S. “Use of transepithelial conductance as a screening technique for
identification of drugs that promote readthrough of premature termination codons.”
Pediatric Pulmonology Supp 36 (Abst 76), 2013.
14. Birket, S; Chu, KK; Li, Y; Houser, GH; Mazur, M; Schuster, BS; Hanes, J; Tearney, GJ;
Rowe, SM. “The relationship between periciliary liquid hydration and mucus transport is
affected by bicarbonate transport.” Pediatric Pulmonology Supp 36 (Abst 85), 2013.
15. Harris, WT; Liu, RM; Kong, M; Halloran, B; Zhang, W; MacEwen, M; Ambalavanan, N;
Sorscher, EJ. “Developing a model of fibrotic lung disease in the CF mouse.” Pediatric
Pulmonology Supp 36 (Abst 184), 2013.
16. Tuggle, KL; Birket, S; Fortenberry, J; Du, M; Tang, LP; Hong, J; Bedwell, DM; Rowe, SM;
Sorscher, EJ; Fanucchi, MV. “Characterization of the CFTR-knockout rat.” Pediatric
Pulmonology Supp 36 (Abst 185), 2013.
17. Hong, JS; Tuggle, KL; Wen, H; Sorscher, EJ. “Constitutive expression of CFTR
suppressor mutations as a tool for investigating F508del correction.” Pediatric
Pulmonology Supp 36 (Abst 195), 2013.
18. Skinner, D; Ranganath, N; Zhang, S; Jones, B; Sorscher, EJ; Woodworth, BA. “Porcine
nasal primary cultures for studies of cystic fibrosis.” Pediatric Pulmonology Supp 36 (Abst
198), 2013.
19. Icyuz, M; McClure, M; Sorscher, EJ; Hartman, JL. “Mutations in YOR1 to model different
aspects of CFTR biogenesis.” Pediatric Pulmonology Supp 36 (Abst 201), 2013.
20. Jackson, L; Tang, LP; Wen, H; Bridges, RJ; Sorscher, EJ; Rowe, SM; Hong, JS.
“Derivation and characterization of a CFTR expressing cell line amenable for ion
transport studies.” Pediatric Pulmonology Supp 36 (Abst 202), 2013.
21. Rowe, SM; Heltshe, SL; Gonska, T; Donaldson, S; Borowitz, D; Gelfond, D; Sagel, SD;
Khan, U; Hamblett, NM; VanDalfsen, J; Joseloff, E; Ramsey, B. “Results of the G551D
observational study: The effect of Ivacaftor in G551D patients following FDA approval.”
Pediatric Pulmonology Supp 36 (Abst 206), 2013.
22. Rowe, SM; Konstan, MW; Accurso, FJ; De Boeck, K; Sermet-Gaudelus, I; Kerem, E;
Wilschanski, M; Miller, NL; Elfring, GL; Spiegel, R; Peltz, S; Barth, J; Ajayi, T. “The use of
chronic inhaled antibiotics in the phase 3 clinical trial of Ataluren in patients with
nonsense-mutation cystic fibrosis.” Pediatric Pulmonology Supp 36 (Abst 207), 2013.
23. Gonska, T; Shamsuddin, AK; Conrad, D; Wine, J; Milla, C; Accurso, FJ; Dupuis, A;
Avolio, J; Mayer Hamblett, N; Heltshe, SL; Joseloff, E; Durie, PR; Quinton, P; Rowe, SM.
“Effect of Ivacaftor on β-adrenergic sweat secretion by evaporimetry in G551D patients.”
Pediatric Pulmonology Supp 36 (Abst 208), 2013.
24. Donaldson, SH; Zeman, K; Laube, B; Corcoran, T; Locke, LW; Pilewski, J; Hanes, J;
Schuster, B; Kanzawa, M; Rowe, SM; Bennett, WD. “Effect of Ivacaftor on mucociliary
clearance and mucus rheology in patients with a G551D CFTR mutation.” Pediatric
Pulmonology Supp 36 (Abst 209), 2013.
25. Sagel, SD; Harris, JK; Wagner, BD; Zemanick, ET; Emmett, P; Taylor-Cousar, JL;
Oermann, CM; Billings, JL; Rubenstein, RC; Rowe, SM. “Effects of Ivacaftor on airway
microbiome and inflammation in G551D patients.” Pediatric Pulmonology Supp 36 (Abst
224), 2013.
26. Liu, B; Hathorne, H; Reeves, GA; Clancy, JP; Rowe, SM. “The role of the Center for
CFTR Detection within the TDN.” Pediatric Pulmonology Supp 36 (Abst 254), 2013.
27. Konstan, MW; Rowe, SM; Accurso, FJ; Kerem, E; Wilschanski, M; De Boeck, K; SermetGaudelus, I; Miller, NL; Elfring, GL; Spiegel, R; Peltz, S; Barth, J; Ajayi, T. “Use of
different pulmonary exacerbation definitions in the phase 3 clinical trial of Ataluren in
patients with nonsense mutation cystic fibrosis.” Pediatric Pulmonology Supp 36 (Abst
258), 2013.
28. Bowers, H; Coricor, G; Birket, S; Li, Y; Wiesmann, W; Baker, S; Clancy, JP; Rowe, SM.
“PAAG improves cystic fibrosis sputum viscosity and elasticity ex vivo.” Pediatric
Pulmonology Supp 36 (Abst 263), 2013.
29. Wilsterman, E; Chu, KK; Li, Y; Diephuis, B; Birket, S; Schuster, BS; Hanes, J; Rowe, SM;
Tearney, GJ. “Rheological validation of particle-tracking micro-optical coherence
tomography.” Pediatric Pulmonology Supp 36 (Abst 267), 2013.
30. Kong, M; Peng, N; Namasivayam, A; Schoeb, T; Sorscher, EJ; Clancy, JP. “A murine
model of cystic fibrosis and respiratory syncytial virus infection.” Pediatric Pulmonology
Supp 36 (Abst 318), 2013.
31. Britton, LJ; Mims, C; Harris, WT; Brown, J. “Predictors of patient outcomes at 12 months
of age in patients diagnosed by newborn screening.” Pediatric Pulmonology Supp 36
(Abst 456), 2013.
32. Hathorne, H; Reeves, G; Tidwell, S; Roberts, T; Tarn, V; Britton, L. “Initiation of EQUIPCR within the clinical research team.” Pediatric Pulmonology Supp 36 (Abst 488), 2013.
33. Self, S; Gunter, L; Hardy, J; Britton, LJ; Kelly, F; Pugh, G; Lachowicz, K; Beech, H;
Gaines, C; Krueger, B. “Family knowledge, utilization and satisfaction of CF psychosocial
team—What do they know, use, and like?” Pediatric Pulmonology Supp 36 (Abst 499),
2013.
34. Anderson, V; Britton, LJ; Troxler, RB; Wooldridge, N; West, K; Shepherd, S. “Does
nutrition knowledge affect BMI for age percentile in children with CF?” Pediatric
Pulmonology Supp 36 (Abst 515), 2013.
35. Gelfond, D; Borowitz, D; Frederick, CA; Uluer, A; Sicilian, L; Konstan, M; Rowe, SM.
“Impact of Ivacaftor therapy on the intestinal pH profile in CF subjects with G551D
mutation.” Pediatric Pulmonology Supp 36 (Abst 540), 2013.
36. Jones, M; Tarn, V; Hoover, W; Britton, LJ; Wooldridge, NH; Oster, R. “Potential risk
factors for low BMI in late adolescent patients with cystic fibrosis.” Pediatric Pulmonology
Supp 36 (Abst 548), 2013.
37. Tuggle, KL; Havasi, V; Sorscher, EJ; Stalvey, MS. “CFTR knockout rats have reduced
growth and IGF-I concentrations.” Pediatric Pulmonology Supp 36 (Abst 577), 2013.
38. Havasi, V; Clines, GA; Stalvey, MS. “Reduced bone density in G551D-CFTR mice: A
novel model to study effects of CFTR on bone metabolism.” Pediatric Pulmonology Supp
36 (Abst 578), 2013.
39. Grossoehme, DH; Szczesniak, R; Dimitriou, S; Britton, LJ; Dodd, C; Quittner, AL; Seid,
M. “Parental adherence: Spiritual, religious and psychosocial influences.” Pediatric
Pulmonology Supp 36 (Abst 605), 2013.
40. Gamble, S; Hoover, W; Britton, LJ; Mims, C; Brown, J; Stodghill, L. “Remediation in
patient/parent knowledge of respiratory therapies.” Pediatric Pulmonology Supp 36 (Abst
651), 2013.
41. Brown, J; Britton, L; Mims, C; Self, S; Troxler, B. “Sexual health: What do adolescents
know?” Pediatric Pulmonology Supp 36 (Abst 658), 2013.
North American Cystic Fibrosis Conference
Atlanta, Georgia
October 9-11, 2014
1. Rowe, SM. “Translational readthrough of premature termination codons in CFTR as a
therapeutic approach to modulate CFTR.” Pediatric Pulmonology Supp 38 (Summary
S16.2), 2014.
2. Stalvey, M. “Basic studies in CF bone disease: lessons from animal models.” Pediatric
Pulmonology Supp 38 (Summary S19.1), 2014.
3. Oliver, KE; Veit, G; Icyuz, M; Guo, J.; McClure, ML; Hong, JS; Wen, H; Sorscher, EJ;
Lukacs, GL; Hartman, JL. “Modulating the dynamics of ribosome function rescues
F508del CFTR maturational arrest.” Pediatric Pulmonology Supp 38 (Abst 5), 2014.
4. Moss, R; Flume, PA; Elborn, J; Cooke, J; Rowe, SM; McColley, SA; Rubenstein, RC;
Pilewski, JM; Higgins, M. “Effects of ivacaftor in CF patients with R117H-CFTR.”
Pediatric Pulmonology Supp 38 (Abst 17), 2014.
5. Liu, L; Park, I; Fan, Y; Bakey, MT; Carver, JR; Kirby, EF; Chettiar, SN; Regan, N; Bhasin,
D; Li, P; Sorscher, EJ; Li, C; Wang, XR. “Initial characterization of NBD1-targeting ∆F508
correctors.” Pediatric Pulmonology Supp 38 (Abst 39), 2014.
6. Hildebrandt, E; Zhang, Q; Cant, N; Ding, H; Dai, Q; Peng, L; Fu, Y; DeLucas, L; Ford, R;
Kappes, J; Urbatsch, IL. “A survey of detergents for improved purification and
concentration of stable, active human cystic fibrosis transmembrane conductance
regulator (CFTR).” Pediatric Pulmonology Supp 38 (Abst 41), 2014.
7. Goeckeler-Fried, J; Chung, W; Chiang, A; Denny, RA; Weissman, A; Sorscher, EJ;
Brodsky, JL. “Inhibitors of CFTR ubiquitination function synergistically with small
molecule correctors to enhance ∆F508-CFTR maturation and activity.” Pediatric
Pulmonology Supp 38 (Abst 50), 2014.
8. He, L; An, J; Brouillette, CG; Riordan, JR. “Development of a high throughput screening
assay for reagents that thermally stabilize CFTR NBD1 using split GFP
complementation.” Pediatric Pulmonology Supp 38 (Abst 53), 2014.
9. Finegan, JF; Harrington, JM; Musisi, I; Bibler, JH; Wong, EI; Liang, F; Hong, JS;
Sorscher, EJ; Mense, M. “Functional classification of CFTR correctors by parallel testing
in two isogenic FRT cell lines expressing CFTR F508del protein with specific suppressor
mutations.” Pediatric Pulmonology Supp 38 (Abst 63), 2014.
10. Zhang, S; Chaaban, M; Skinner, D; Birket, SE; Rowe, SM; Sorscher, EJ; Woodworth, BA.
“Resveratrol overcomes transepithelial fluid and electrolyte imbalance in a hypoxiainduced model of acquired CFTR deficiency.” Pediatric Pulmonology Supp 38 (Abst 64),
2014.
11. Bali, V; Lazrak, A; Guroji, P; Matalon, S; Bebok, Z. “The I507-ATC→ATT silent codon
change hinders corrector-mediated rescue of ∆F508 CFTR.” Pediatric Pulmonology
Supp 38 (Abst 65), 2014.
12. Wang, C; Aleksandrov, AA; Proctor, EA; Kota, P; Yang, Z; An, J; Forouhar, F; Boel, G;
Riordan, JR; Dokholyan, NV; Brouillette, CG; Hunt, JF. “Nucleotide analogs with
modestly enhanced binding affinity for NBD1 provide significant correction of the
temperature-dependent defect in F508del-CFTR.” Pediatric Pulmonology Supp 38 (Abst
70), 2014.
13. Sabusap, C; Hong, JS; McClure, M; Chung, W; Wen, H; Sorscher EJ. “Impact of
palmitoylation on clinically significant mutations characterized by CFTR2.” Pediatric
Pulmonology Supp 38 (Abst 76), 2014.
14. Solomon, GM; Frances, R; Chu, KK; Gabriel, G; Birket, SE; Lemke, K; Klena, N;
Mojahed, D; Tearney, G; Lo, C; Rowe, SM. “Micro-OCT to interrogate mechanisms of
altered ciliary dynamics and mucus transport.” Pediatric Pulmonology Supp 38 (Abst 83),
2014.
15. Grayson, J; Zhang, S; Skinner, D; Fortenberry, J; Sorscher, EJ; Woodworth, BA. “In vitro
and in vivo models of acute inflammation demonstrate acquired defects in transepithelial
CL transport.” Pediatric Pulmonology Supp 38 (Abst 86), 2014.
16. Birket, SE; Tuggle, KL; Chu, KK; Tearney, GJ; Fanucchi, MV; Sorscher EJ; Rowe, SM.
“CFTR-/- rat exhibits delayed mucociliary clearance characteristic of cystic fibrosis airway
disease.” Pediatric Pulmonology Supp 38 (Abst 87), 2014.
17. Raju, S; Trombley, JE; Kim, H; Birket, SE; Lin, VY; Samuel, SL; Tang, L; Warren, M;
Winter, L; Dohm, E; Cadillac, JM; Zinn, KR; Schoeb, TR; Rowe, SM. “Acquired CFTR
dysfunction in a novel ferret model of COPD.” Pediatric Pulmonology Supp 38 (Abst 88),
2014.
18. Ford, TN; Chu, KK; Birket, SE; Solomon, GM; Mazur, M; Rowe, SM; Tearney, GJ. “Dualmodality fluorescence/micro-optical coherence tomography.” Pediatric Pulmonology
Supp 38 (Abst 94), 2014.
19. Szul, T; Bratcher, PE; Kong, M; Xu, X; Gaggar, A. “Defective CFTR airway epithelial cells
secrete exosomes containing prolyl endopeptidase.” Pediatric Pulmonology Supp 38
(Abst 141), 2014.
20. Crane, AM; Kramer, P; Bui, JH; Chung, WJ; Li, XS; Gonzalez-Garay, ML; Hawkins, F;
Liao, W; Mora, D; Wang, J; Sun, HC; Paschon, DE; Guschin, DY; Gregory, PD; Kotton,
DN; Holmes, MC; Sorscher, EJ; Davis, BR. “Sequence-specific genetic correction and
restored function of CFTR gene in cystic fibrosis induced pluripotent stem cells.”
Pediatric Pulmonology Supp 38 (Abst 165), 2014.
21. Hong, JS; Sorscher, EJ; Plyler, ZE; Keiles, S. “Defining allelic heterogeneity among
clinically important mutations in CFTR.” Pediatric Pulmonology Supp 38 (Abst 170),
2014.
22. Hong, JS; Mahiou, J; Liang, F; Bihler, JH; Mense, M; Lukacs, GL; Wen, H; Sorscher, EJ.
“Epithelial models encoding diverse CFTR2 mutations for studies of disease mechanism
and drug discovery.” Pediatric Pulmonology Supp 38 (Abst 175), 2014.
23. Skinner, D; Zhang, S; Dunlap, Q; Tuggle, KL; Sorscher, EJ; Woodworth, BA. “Ion
transport phenotype of upper airway epithelium in the CFTR-/- rat.” Pediatric
Pulmonology Supp 38 (Abst 179), 2014.
24. Harris, WT; Jilling, T; Halloran, B; Zhang, W; MacEwen, M; Ambalavanan, N; Sorscher,
EJ. “Pulmonary remodeling pathways in human CF and the porcine model.” Pediatric
Pulmonology Supp 38 (Abst 181), 2014.
25. Plyler, ZE; Fanucchi, MV; Tuggle, KL; Pierucci-Alves, F; Harris, WT; Schultz, BD;
Sorscher, EJ. “Involution of vas deferens as a model for epithelial disruption in the CF
rat.” Pediatric Pulmonology Supp 38 (Abst 188), 2014.
26. Heltshe, SL; Mayer-Hamblett, N; Burns, JL; Khan, U; Baines, A; Ramsey, B; Rowe, SM.
“Ivacaftor is associated with pseudomonas aeruginosa reduction in cystic fibrosis patients
with G551D-CFTR.” Pediatric Pulmonology Supp 38 (Abst 195), 2014.
27. Wells, JM; Farris, RF; Dransfield, MT; Rowe, SM. “CT-detected pulmonary artery
enlargement predicts pulmonary exacerbations in cystic fibrosis.” Pediatric Pulmonology
Supp 38 (Abst 213), 2014.
28. Mutyam, V; Liu, B; Du, M; White E; Bostwick, R; Rasmussen, L; Tower, N; Bedwell, DM;
Rowe, SM. “A screen to identify clinically available agents that promote suppression of
premature termination codons.” Pediatric Pulmonology Supp 38 (Abst 232), 2014.
29. Solomon, GM; Liu, B; Sermet-Gaudelus, I; Fajac, I; Wilschanski, M; Rowe, SM.
“Comparative statistics demonstrate consistency of multiple readers of NPD parameters
using an NPD scoring algorithm.” Pediatric Pulmonology Supp 38 (Abst 242), 2014.
30. Bratcher, PE; Rowe, SM; Szul, T; Harris, WT; Tirouvanziam, R; Gaggar, A. “Ivacaftor
treatment results in modulation of blood leukocyte activation in cystic fibrosis patients
with the G551D CFTR mutation.” Pediatric Pulmonology Supp 38 (Abst 246), 2014.
31. Pace, J; Stalvey, MS; Tarn, V; Heltshe, SL; Rowe, SM. “Ivacaftor improves linear growth
in G551D pre-pubertal children.” Pediatric Pulmonology Supp 38 (Abst 252), 2014.
32. Fernandez, CM; Watson, JD; Bowers, H; Chu, KK; Li, Y; Birket, SE; Mazur, M; Raju, S;
Tearney, GJ; Baker, S; Rowe, SM. “PAAG decreases viscosity and improves
transportability of cystic fibrosis mucus in situ.” Pediatric Pulmonology Supp 38 (Abst
253), 2014.
33. Rowe, SM; McColley, SA; Rietschel, E; Li, X; Bell, SC; Konstan, M; Marigowda, G; Waltz,
D; Boyle, MP; VX09-809-102 Study Group. “Effect of 8 weeks of lumacaftor in
combination with ivacaftor in patients with CF and heterozygous for the F508del-CFTR
mutation.” Pediatric Pulmonology Supp 38 (Abst 254), 2014.
34. Xue, X; Brooke, D; Mutyam, V; Du, M; Prevelige, P; Rowe, SM; Bedwell, DM.
“Identification and functional consequences of the alternate amino acids inserted during
suppression of the CFTR-G542X nonsense mutation.” Pediatric Pulmonology Supp 38
(Abst 255), 2014.
35. Konstan, M; Rowe, SM; Accurso, FJ; Kerem, E; Wilschanski, M; Sermet-Gaudelus, I;
DeBoeck, K; Elfring, G; Spiegel, R; Peltz, S; Ajayi, T. “Sustained improvement in lung
function in an open-label extension study of Ataluren in patients with nonsense mutation
cystic fibrosis.” Pediatric Pulmonology Supp 38 (Abst 266), 2014.
36. DeBoeck, K; Sermet-Gaudelus, I; Kerem, E; Wilschanski, M; Accurso, FJ; Konstan, M;
Rowe, SM; Elfrign, G; Spiegel, R; Peltz, S; Ajayi, T. “Design of the Ataluren confirmatory
Phase 3, randomized, double-blind, placebo-controlled trial in patients with nonsense
mutation cystic fibrosis (ACT CF).” Pediatric Pulmonology Supp 38 (Abst 269), 2014.
37. Xu, X; Abdalla, T; Sabbatini, G; Roberts, T; Bratcher, P; Jackson, PL; Blalock, JE;
Clancy, JP; Gaggar, A. “A randomized double-blinded placebo controlled trial of
doxycycline as an adjunctive therapy for the treatment of inpatient cystic fibrosis
exacerbation.” Pediatric Pulmonology Supp 38 (Abst 270), 2014.
38. Chu, KK; Unglert, C; Carruth, RW; Ford, TN; Singh, K; Birket, SE; Solomon, GM; Rowe,
SM; Tearney, GJ. “In vivo functional imaging of cilia and mucociliary transport with high
resolution µOCT.” Pediatric Pulmonology Supp 38 (Abst 278), 2014.
39. Vu, E; Sorscher, EJ; Lowery, R; Hayes, S. “An HTS-compatible plate for highly
miniaturized cultures of primary human bronchial epithelial cells at air-liquid interface.”
Pediatric Pulmonology Supp 38 (Abst 279), 2014.
40. Nichols, D; Donaldson, SH; Dorkin, H; Flume, PA; Gaggar, A; Chmiel, J; Griffin, R. “A 3
week dose escalation, randomized, double-blind, placebo-controlled trial to assess the
safety, tolerability, and possible efficacy of 100 mg or 200 mg of once daily inhaled
Alpha-1 HC in cystic fibrosis (CF).” Pediatric Pulmonology Supp 38 (Abst 281), 2014.
41. Kong, M; Peng, N; Szul, T; Jackson, PL; Blalock, E; Gaggar, A. “RSV infection leads to
activation of the MMP-9/PGP cascade in vivo.” Pediatric Pulmonology Supp 38 (Abst
374), 2014.
42. Lauderdale, SE; Britton, LJ; Rodgers, TF. “IGE screening in patients with cystic fibrosis.”
Pediatric Pulmonology Supp 38 (Abst 472), 2014.
43. Carden, JL; Antony, V. “Implementation of FEV1 action guide results in greater than
twelve percent mean FEV1 increase in adult cystic fibrosis patients 18 years and older.”
Pediatric Pulmonology Supp 38 (Abst 486), 2014.
44. Havasi, V; Tuggle, KL, Birket, SE; Sorscher, EJ; Stalvey, MS. “Characterization of CFTRrelated bone disease in the CF rat model.” Pediatric Pulmonology Supp 38 (Abst 560),
2014.
45. Havasi, V; Clines, GA; Stalvey, MS. “Impact of G551D-CFTR on bone metabolism in the
murine model.” Pediatric Pulmonology Supp 38 (Abst 561), 2014.
46. Woodall, MT; Thrasher Self, S; Harris, WT. “Caregiver response to CF carrier
identification in the Alabama CF NBS program.” Pediatric Pulmonology Supp 38 (Abst
627), 2014.
47. Mims, C; Britton, LJ; Dunn, J; Harris, WT. “Does nursing intervention impact parental
decision to quit smoking?” Pediatric Pulmonology Supp 38 (Abst 631), 2014.
48. Jackson, TF; Troxler, B; Mims, C; Gamble, S; Britton, LJ. “Clinical pharmacist
assessment of the use of education tools in patient knowledge and adherence to
respiratory medications.” Pediatric Pulmonology Supp 38 (Abst 639), 2014.