BIOGRAPHICAL SKETCH
NAME:
Jan A. Nolta, Ph.D.
eRA COMMONS USER NAME:
POSITION TITLE:
Jnolta
Professor, Director of the Stem Cell Program,
University of California, Davis
EDUCATION/TRAINING (Begin with baccalaureate or other initial professional education, such as nursing, and include postdoctoral training.)
INSTITUTION AND LOCATION
California State University Sacramento, CA
University of Southern California, L.A., CA
Childrens Hospital of Los Angeles, CA
DEGREE
B.S.B.S.
Ph.D.
Ph.D.
Post-Doc
Postdoc
YEAR(s)
1984
1984
1984
1990-94
1994
1994-97
1996
FIELD OF STUDY
Biology
Biology
Biology
Molecular
Microbiology
Microbiology
Stem
Gene
Post-Doc
Cell
Therapy
Gene 1996
Therapy
Personal Statement:
I am the Director of the Stem Cell Program at UC Davis School of Medicine, and I direct the Institute for Regenerative
>
Cures.
The UC Davis stem cell program has over 150 faculty members collaborating to work toward stem cell-related
cures for a spectrum of diseases and injuries. The current research in our laboratory is focused on developing therapies
that will use stem cells to deliver factors for treating disease and tissue injury. Our group focuses on “bench to the
bedside” research, and I have been involved in numerous clinical trials of gene and cell therapy. I have more than 25
years’ experience with human stem cells, have published over 100 manuscripts in the stem cell field and have authored 30
book chapters. I have served on over 200 review panels for the National Institutes of Health and other grant-funding
agencies, am Editor for the Journal “Stem Cells” and editor of the Book "Genetic Engineering of Mesenchymal Stem
Cells". In 2012 I received a prestigious Transformative Grant Award from the NIH office of the Director, based on our
novel work on cell-to-cell communication. My passion is to help our teams bring exciting advances in stem cell therapy
from the bench to the bedside, to develop novel clinical trials of regenerative medicine to help our patients who have few
other options.
Professional Experience and Duties:
Professor, Director of the Stem Cell Program and Institute for Regenerative Cures, Scientific Director-GMP Facility for
Cell and Gene Therapy, UC Davis School of Medicine, Department of Internal Medicine, Division of Hematology/
Oncology. November 1, 2006 - present.
Associate Professor of Medicine & Pathology/Immunology, Washington University School of Medicine, Div. Oncology,
- Stem Cell Program (May 2002 –December 2006). Tenure granted September 2002.
Scientific Director, Washington University Good Manufacturing Practice (GMP) Facility (December 2002 to Feb 07).
Washington Univ. Animal Studies Committee (2003- 2006) Member, CHLA Animal Care Committee (1997 – 2002)
Assistant Professor of Pediatrics, Univ. of Southern California School of Medicine, L.A., CA (June 1997- May 2002)
Member, Norris Comprehensive Cancer Center, Developmental Therapeutics & Clin. Trials Research program (1998-02)
Chair, Institutional Biosafety Committee, Childrens Hospital of Los Angeles (May 2000 – May 2002)
Graduate Faculty, USC School of Medicine, Department of Developmental Biology (July 1997-March 2002)
Postdoctoral Fellow, John Connell Gene Therapy Foundation –Donald Kohn’s laboratory, CHLA (1994-1997)
Graduate Student, University of Southern California School of Medicine, Dept. of Molecular Microbiology (1990 – 1994)
Research Specialist II-IV, Dept. Research Immunology/ BMT, Childrens Hospital Los Angeles, L.A., CA (1987-1990)
Hematology Laboratory Technician/ Night Shift Supervisor, UC Davis Medical Center (1983-87)
Honors:
Honors at Entrance, California State University; Dean's Honor List, CSUS, UC Davis, USC
Lifetime Member California Scholarship Foundation (1980), BA Scholarship (‘83), Golden Key Honor Society (‘84)
American Society of Hematology Travel Grants (1988-1995), NRSA Training Grant (1991 - 1993)
Dean's Honor List, USC Graduate School (4.0 GPA; 1990 - 1994)
International Society of Hematology New Investigator Award (1996)
Research Career and Development Award, CHLA Research Institute (1996)
James Shannon Directors Award, NIH NIDDK (1997)
Huntington’s Disease Society of America Distinguished Leadership Award (2008)
California State University, Sacramento Distinguished Service Award (2009)
Deans Award for Team Excellence (2011), Deans Award for Excellence in Mentoring (2013)
Elected: AAAS Fellow (2013)
Top 50 most influential global stem cell scientists (2013)
Activities:
Member: American Society of Hematology, AAAS, International Society of Experimental Hematology; American
Society for Gene Therapy, International Society for Stem Cell Research (ISSCR)
Gene T
International Society for Hematology and Graft Engineering (ISHAGE) Gene Therapy Committee (1998-99)
Moderator/ reviewer/session chair at the Annual Meeting of the American Society for Hematology (1997 – 2009)
P01 review panels & site visits, NHLBI, NCI, NIDDK 1999-2007, Chair NIDDK, NHLBI stem cell reviews 2003,
Reviewer, Italian Telethon Grants Administration Program, 1998 – present, NIH NCRR COBRE grants 2008
US Army BCA grant review sessions, 2000, 2001.Chair: IBC Gene Therapy Workshop, Lake Tahoe, CA, 1999
External Advisory Boards: Univ. Minnesota Stem Cell PPG (2000-2002) & Seattle CF Gene Therapy Program, 2000
Ad-hoc member, HEM-1 and HEM-2 Study Sections, NIH (1998- 2003)
International Society of Experimental Hematology (ISEH) Nominating Committee, 2001-4. Chair: 2003-4.
American Society of Gene and Cell Therapy, Hematopoietic Therapy committee, 2003-2006. Chair, 2004-2006,
Chair, ASGT embryonic stem cell and tissue engineering committee, 2006-2009, nominating committee 2009-.
Editorial Board, Human Gene Therapy, 2001-2009, Editorial Board, Experimental Hematology, 2000 – present
Section Editor, Leukemia: stem cells, 2001 – 2002, Gene Therapy 2000-2006. Editorial Board,1999-2005.
Editorial Board, Blood, September 2002 – 2008, Editorial Board, Molecular Therapy, 2003- 2006.
Full Member, Hematology B Study Section, National Institutes of Health, May 2003-June 2007. Ad Hoc 2008Chair, New York Stem Cell Research Program (NYSTEM) Major Shared Facilities Panel, 2008 - 2010
Member, Huntington’s disease Communications Group of the Huntington’s Study group, 2008-present
Member, International Society of Cell Therapy Commercialization Committee, 2011-present
NHLBI Production Assistance for Cellular Therapies (PACT) External Expert Panel Member, 2010-present
Associate Editor, Stem Cells, February 2008 – December 2011
Editor-in-Chief, Stem Cells, January 2012 - 2016
Publications (Selected, of over 150 manuscripts)
1. Barranger JA, Ohashi T, Hong CM, Tomich J, Aerts JF, Tager JM, Nolta JA, Sender LS, Weiler S, Kohn DB.
Molecular pathology and therapy of Gaucher disease. Journal of Inherited Metabolic Disease 51:45-71, 1989.
2. Nolta JA, Kohn DB. Expression of human Glucocerebrosidase in murine Long Term Bone Marrow Cultures after
retroviral vector-mediated transfer. Blood 75:787-97, 1990.
3. Ozkaynak M, Nolta JA, Parkman R. Purging of rhabdomyosarcoma cells using 4-HC. Cancer Research 50:1455-58,
1990.
4. Nolta JA, Kohn DB. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the
proliferative status of human hematopoietic progenitor cells. Human Gene Therapy 1(3):257-268, 1990.
5. Lenarsky C, Weinberg K, Nolta J, Annett G, Kohn D, Parkman R. Autologous bone marrow transplantation with 4HC purged marrows for children with acute non-lymphoblastic leukemia in 2nd remission. Bone Marrow
Transplantation 6:425-429, 1990.
6. Weinthal J, Nolta JA, Yu X-J, Lilley J, Uribe L, Kohn D. Expression of human Glucocerebrosidase following
retroviral vector-mediated transduction of murine hematopoietic stem cells. Bone Marrow Transplantation 8:403412, 1991.
7. Kohn D, Nolta J, Yu X-J, Lilley J, Crooks G. Towards gene therapy for Gaucher disease. Human Gene Therapy
2:101-105, 1991.
8. Tishler DM, Weinberg KI, Sender LS, Nolta JA, Raffel C. Multidrug resistance gene expression in pediatric
primitive neuroectodermal tumors of the central nervous system. Journal of Neurosurgery 76:507-512, 1992.
9. Nolta JA, Crooks GM, Overell RW, and Kohn DB. Retroviral vector-mediated gene transfer into primitive human
hematopoietic progenitor cells: effects of mast cell growth factor (MGF) combined with other cytokines. Expt.
Hematology 20:1065-1071, 1992.
10. Nolta JA, Yu XJ, Kohn DB. Retroviral-mediated transfer of the human Glucocerebrosidase gene into Gaucher BM.
J. Clin. Invest. 90:342-348, 1992.
11. Nolta JA, Hanley M, Kohn DB. Sustained human hematopoiesis in immunodeficient mice by co-transplantation of
marrow stroma expressing human IL-3: Analysis of gene transduction of long-lived progenitors. Blood 83:30413051, 1994.
12. Nolta JA, Smogorzewska EM, Kohn DB. Analysis of optimal conditions for retroviral-mediated transduction of
primitive human hematopoietic stem cells. Blood 86; 101 - 110, 1994.
13. Hanley ME, Nolta JA, Parkman R, and Kohn DB. Umbilical cord blood cell transduction by retroviral vectors:
preclinical studies to optimize gene transfer. Blood Cells 20; 539 - 546, 1994.
14. Wells S, Kohn D, Nolta JA. Stromal support optimizes transduction of Gaucher LTCIC. Gene Therapy 2, 512-520,
1994.
15. Kohn DB, Weinberg KI, Nolta JA., Parkman R. et al. Engraftment of gene-modified cells from Umbilical Cord
Blood in neonates with Adenosine DeAminase (ADA) deficiency. Nature Medicine 1; 1017-1023, 1995.
16. Nolta JA, Dao MA, and Kohn DB. Transduction of pluripotent human hematopoietic stem cells demonstrated by
clonal analysis after engraftment in immune deficient mice. Proc. Natl. Acad. Sci. USA 93; 2414-2419, 1996.
17. Dao MA and Nolta JA. Inclusion of IL-3 During Retroviral-Mediated Transduction on Stromal Support Does
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Not Increase the Extent of Gene Transfer Into Long-Term Engrafting Human stem cells. Cell. Mol. Ther. 3:8190, 1997.
Dao MA, Hannum CH, and Nolta JA. FLT3 ligand preserves the ability of human CD34+ progenitors to sustain
long-term hematopoiesis in immune deficient mice following ex-vivo retroviral - mediated transduction. Blood
89:446-456, 1997.
Nolta JA, Kohn DB. Hematopoietic stem cells for gene therapy. In Stem Cells, Ed: C. Potten, Academic Press, pp.
447-61, 1997.
Dao MA, Yu XJ, and Nolta JA. Clonal diversity of retroviral vector - marked primitive human hematopoietic
progenitors recovered from long-term engrafted immune deficient mice. Expt. Hematol. 25:1357-1366, 1997.
Dao MA, Pepper, KA, and Nolta JA. Human cytokine production from engineered stromal cells influences
hematopoietic development from co-transplanted human CD34+ progenitors in immune deficient mice. Stem Cells
15:443-453, 1997.
Dao MA, Hashino K, Kato I, and Nolta JA. Adhesion to fibronectin maintains regenerative capacity during ex-vivo
culture and transduction of human hematopoietic stem and progenitor cells. Blood 92: 4612-4621, 1998.
Dao M, Nolta JA. Hematopoietic stem cells as targets for Gene Therapy: Cancer Research, Therapy and Control
7:19-25, 1998.
Dunbar CE, Kohn DB, Schiffmann R, Barton NW, Nolta JA, Esplin J, Pensiero M, Emmons RVB, Leitman S, Krebs
CB, Carter CC, Brady RO, and Karlsson S. Retroviral transfer of the glucocerebrosidase gene into CD34+ cells from
patients with Gaucher disease: in vivo detection of transduced cells without myeloablation. Human Gene Therapy 9:
2692-2640, 1998.
Dao MA, Shah AJ, Smogorzewska EM, Crooks GC, and Nolta JA. Comparison of human hematopoietic progenitor
content and retroviral marking in immune deficient mice transplanted with CD34+ vs. CD34+/38- cells. Blood 91(3):
1243-1255, 1998.
Kohn DB, Hershfeld MS, Nolta JA, Parkman R et al. Selective accumulation of T cells containing a normal ADA
gene four years after transduced autologous UCB CD34+ cell BMT in ADA-deficient SCID neonates. Nature
Medicine 4:775-780, 1998.
Dao M, Taylor N, Nolta J. Reduction in levels of the CDK inhibitor p27kip-1
alization
induces cell cycle entry & increases retroviral transduction of primitive human hematopoietic cells. Proc Natl Acad
Sci USA 95:13006-13011, 1998.
Epstein A, Liu M, Nolta JA, and Durden DL.
g in
primary human macrophages. Journal of Leukocyte Biology, 65: 1-12, 1999.
Dao MA and Nolta JA. Molecular control of cell cycle progression in primary human hematopoietic stem cells:
Methods to increase levels of retroviral - mediated transduction. Leukemia 13:1473-1480, 1999.
Arakawa-Hoyt J, MA Dao, F Thiemann, KI Weinberg, GM Crooks, and Nolta JA. The number and generative
capacity of human B cell progenitors is higher in umbilical cord blood than in bone marrow. Bone Marrow
Transplantation 24: 1167-1176, 1999.
Dao MA, Tsark E, Nolta JA. Animal xenograft models for evaluation of gene transfer into human hematopoietic stem
cells. Current Opinion in Molecular Therapeutics 1:553-557, 1999.
Parkman R, Weinberg K, Crooks G, Nolta J, Kohn D. Gene therapy for ADA deficiency. Ann. Rev. Med. 51:33-47,
2000.
Evans M, Jordan C, Nolta, JA, Wivel N et al. Clinical Infection Control in Gene Therapy. Infection Control 21: 659 –
673, 2000.
Tsark E, Dao MA, Weinberg KI, and Nolta JA. Interleukin 7 (IL-7) enhances the responsiveness of human T cells
that develop in the bone marrow of athymic mice. Journal of Immunology 166: 170 – 181, 2001.
Wang X, Dao MA, Kuo I and Nolta JA. Phenotypic comparison of extrathymic human bone marrow-derived T cells
with thymic-selected T cells recovered from different tissues. Clinical Immunology, 100 (3), 339 - 348, 2001.
Nolta JA, Thiemann F, Arakawa J, Dao M, Barsky, Moore KA, Lemischka I, Crooks GM. The AFT024 stromal cell
line supports long-term ex vivo maintenance of engrafting multipotent human hematopoietic progenitors. Leukemia
16: 352-361, 2002.
Nolta JA. Cell - Based Therapy: will it replace pharmaceuticals in some cases? Current Drugs 4:212-218, 2002
Dao MA., Hwa J and Nolta JA. Molecular mechanism of transforming growth factor beta-mediated cell-cycle
modulation in primary human CD34(+) progenitors. Blood. Jan 15; 99 (2):499-506, 2002.
Gussoni E, Meyerrose TE, Nolta JA, Weinberg KA, Kunkel LM, et al. Long-term persistence of donor nuclei in a
Duchenne muscular dystrophy patient receiving bone marrow transplantation. Journal of Clinical Investigation Sept.;
110(6):807-14, 2002.
Dao MA, Arevalo JA, and Nolta JA. Reversibility of CD34 expression on human hematopoietic stem cells that retain
the capacity for secondary reconstitution. Blood Jan 1; 101(1); 112-118, 2003.
41. Wu G, Nolta JA, Starnes V, and Cramer D. Migration of mesenchymal stem cells to heart allografts during chronic
rejection. Transplantation Mar 15; 75(5): 679-685, 2003.
42. Bauer G, Nolta JA, Walker JE, Devine S, DiPersio J. Design and construction of a GMP facility for cellular and gene
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therapy in an academic center-implementing new regulatory requirements. Stem Cell and Cellular Therapy 1:54-61,
2003.
Schmidt M, Carbonaro D, Speckmann C, Bohnsack J, Nolta JA, Kohn D, von Kalle C. Clonality Analysis after
retroviral–mediated gene transfer to cord blood CD34+ cells of an ADA-deficient SCID infant. Nature Medicine,
Apr1; 9(4); 463-68, 2003.
Wang X, Ge S, McNamara G, Hao QL, Crooks GM, and Nolta JA. Albumin-expressing hepatocyte-like cells develop
in the livers of immune-deficient mice transplanted with highly purified human stem cells. Blood, May 15; 101 (10):
4201-4208, 2003.
Kohn DB, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Blau CA, Sorrentino B, Nolta J, et al. American
Society of Gene Therapy ad hoc subcommittee on retroviral-mediated gene transfer to stem cells. Mol Ther.
Aug;8(2):180-7, 2003
Mohamedali A, Moreau-Gaudry F, Richard E, Xia P, Nolta J, Malik P. Self-inactivating lentiviral vectors resist
proviral methylation but do not confer position-independent expression in hematopoietic stem cells. Mol Ther.
Aug;10(2):249-59, 2004.
J Bonde, D Hess, J Nolta. Recent advances in hematopoietic stem cell biology. Curr Opin Hematol. Nov;11(6):3928, 2004.
D Hess, T Meyerrose, L Wirthlin, M Creer, P Herrbrich, T Craft, Nolta J. Functional characterization of highly
purified human hematopoietic repopulating cells isolated based on aldehyde dehydrogenase activity. Blood,
Sept.104:1648-55, 2004.
Smith E, Meyerrose T, Kohler T, Nolta JA, Muller R, Bab I, Frenkel B. Leaky ribosomal scanning in mammalian
genomes: significance of histone H4 alternative translation in vivo. Nucleic Acids Res. Mar 01;33(4):1298-308, 2005.
Sohara Y, Shimada H, Minkin C, Erdreich-Epstein A, Nolta JA, DeClerck YA. Bone marrow mesenchymal stem
cells provide an alternate pathway of osteoclast activation and bone destruction by cancer cells. Cancer Res. Feb 15;
65: 1129-35, 2005.
Hess DA, Wirthlin L, Craft T, Hohm S, Lahey R, Eades WC, Creer MH, Nolta JA. Selection based on CD133 and
high aldehyde dehydrogenase activity isolates long-term reconstituting human hematopoietic stem cells. Blood,
March 1; 107(5):2162-9, 2006.
Dao MA and Nolta JA. Cytokine and integrin stimulation synergize to promote higher levels of GATA-2, c-myb, and
CD34 protein in primary human hematopoietic progenitors from bone marrow. Blood.109(6):2373-9, Mar 15, 2007.
Meyerrose TE, DeUgarte DA, Hofling A, Herrbrich P, Sands MS, Hedrick MA, and Nolta JA. In vivo Distribution of
Adipose-Derived Mesenchymal Stem Cells in Novel Xenotransplant Models. Stem Cells.; 25; 220-227, 2007.
Partlow KC, Brant J, Chen J, Meyerrose TE, Nolta JA, Caruthers S, Lanza G, and Wickline SA. 19F magnetic
resonance imaging for stem/progenitor cell tracking with multiple unique perfluorocarbon nanobeacons. FASEB J.
Jun;21(8):1647-54, 2007.
Hess DA, Bonde J, Wirthlin L, Dipersio J, Devine S, Nolta J. Human progenitors rapidly mobilized by AMD3100
repopulate NOD/SCID mice with increased efficiency compared to G-CSF-mobilized cells. Biol.Blood
MarrowTransplnt 13:398-411, 2007.
Dao MA, Nolta JA, Creer MH, Verfaillie CM. Biology of umbilical cord blood progenitors in bone marrow niches.
Mar 19. Blood Jul 1;110(1):74-81, 2007.
S X. Qiu, C Dan, L-S Ding, S Peng, S-N Chen, N Farnsworth, J Nolta, M Gross, Ping Zhou. A Triterpene Glycoside
from Black cohosh that Inhibits Osteoclastogenesis by Modulating RANKL and TNF Signaling Pathways. Chem
Biol. 14:860-9, 2007.
B. Nervi, M. Rettig, J. Ritchey, J. Nolta, G. Bauer, J. DiPersio. A new model for GvHD: naïve and ex-vivo activated
human T cells generate consistent engraftment and lethal disease in NOD/SCID/B2M null mice. Exp Hem.
Dec;35(12):1823-38, 2007.
D. Maxwell, J. Bonde, D. Hess, M. Creer, R. Lahey, D. Piwnica-Worms, and J. Nolta. Fluorophore-conjugated iron
oxide nanoparticle labeling and analysis of engrafting human stem cells. Stem Cells. Feb;26(2):517-24. 2008.
D. Hess, T. Craft, L. Wirthlin, W. Eades, M. Creer, M. Sands, J. Nolta. Widespread nonhematopoietic tissue
distribution by transplanted human progenitor cells with high aldehyde dehydrogenase activity. Stem Cells.
Mar;26(3):611-20. 2008.
G Bauer, M Dao, S Case, P Herrbrich, J Arevalo, T Meyerrose, X Wang, S Csik, D Skelton, D B. Kohn, and J Nolta.
In Vivo Biosafety Model to Assess Risk of Adverse Events from Retroviral and Lentiviral Vectors. Mol Ther.
Jul;16(7):1308-15, 2008.
62. T Meyerrose, M Roberts, K Ohlemiller, C Vogler, L Wirthlin, J Nolta, M Sands. Lentiviral-transduced human
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mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplant model of human
disease. Stem Cells.26:1713-22. 2008.
Zhou P, Hohm S, Capoccia B, Link D, and Nolta JA. Immunodeficient mouse models to study human stem-cell
mediated repair of tissue injury. Methods Mol Biol; 430:213-25, 2008.
J.Rich, I. Rosova, J. Nolta, T. Myckatyn, L. Sandell, A. McAlinden. Upregulation of Runx2 and Osterix during in
vitro chondrogenesis of human adipose-derived stromal cells. Biochem Biophys Research Com. Jul 18;372(1):230-5.
2008.
Rosová I, Dao MA, Capoccia BJ, Link DC, Hess DA, Nolta JA. Hypoxic Preconditioning Results in Increased
Motility and Improved Therapeutic Potential of Human Mesenchymal Stem Cells. Stem Cells. Aug;26(8):2173-82.
2008.
Anderson JS, Javien J, Nolta JA, Bauer G. Preintegration HIV-1 Inhibition by a Combination Lentiviral Vector
Containing a Chimeric TRIM5alpha Protein, a CCR5 shRNA, and a TAR Decoy. Mol Ther. 17(12):2103-14. 2009.
Anderson J, Walker J, Nolta JA, Bauer G. Specific Transduction of HIV-Susceptible Cells for CCR5 Knockdown and
Resistance to HIV Infection: A Novel Method for Targeted Gene Therapy and Intracellular Immunization. J Acquir
Immune Defic Syndr. Oct 1;52(2):152-61. 2009.
P. Zhou, L. Wirthlin, J. McGee, G. Annett, and J. Nolta. Contribution of human hematopoietic stem cells to liver
repair. Seminars in Immunopathology Sep;31(3):411-9. Epub 2009 Jun 17.2009.
P. Zhou, R. Lahey, S. Hohm, D. Hess, J Nolta. Human progenitor cells with high aldehyde dehydrogenase activity
efficiently engraft into damaged liver in a novel model. Hepatology 49 (6): 1992 – 2000, 2009.
Capoccia BJ, Robson DL, Levac KD, Maxwell DJ, Hohm SA, Neelamkavil MJ, Bell GI, Xenocostas A, Link DC,
Piwnica-Worms D, Nolta JA, Hess DA. Revascularization of ischemic limbs after transplantation of human bone
marrow cells with high aldehyde dehydrogenase activity. Blood 21 May, 113 ( 21): 5340-5351, 2009.
P Zhou, S Gross, J-H Liu, B-Y Yu, LL Feng, JA Nolta, V Sharma, D Piwnica-Worms and SX Qiu. Flavokawain B,
the Hepatotoxic Constituent from Kava Root, Induces GSH-Sensitive Oxidative Stress through Modulation of
IKK/NF-κB and MAPK Signaling Pathways. FASEB J. Dec;24(12):4722-32, 2010.
I. Rosová, D. Link, and Nolta J. Small Interfering RNA-Mediated Decreases in c-Met Levels Affect the
Differentiation Potential of Human Mesenchymal Stem Cells and Reduce Their Capacity for Tissue Repair. Tissue
Engineering Aug;16(8):2627-39, 2010.
Sondergaard CS, Hodonsky CJ, Khait L, Shaw J, Sarkar B, Birla R, Bove E, Nolta J, Si MS. Human Thymus
Mesenchymal Stromal Cells Augment Force Production in Self-Organized Cardiac Tissue. Ann Thorac Surg. Sep;
90(3):796-804, 2010.
C Sondergaard, D Hess, D Maxwell, I Rosová, D Piwnica-Worms, A Kovacs, L Pedersen, and J Nolta. Human UCB
Progenitors with High ALDH Activity Improve Vascular Density in Acute Myocardial Infarction. J. Translational
Medicine. 8:24-9, 2010.
Zhou P, Estrada, D, Lingala S, Zern M, Nolta, J, Wu, J. Decellularized Liver Matrix as a Carrier for Transplantation
of Human Fetal and Primary Hepatocytes in Mice. Liver Transplantation; 17(4):418-27. 2010.
Joyce N, Annett G, Wirthlin L, Olson S, Bauer G, Nolta J. Mesenchymal stem cells for the treatment of
neurodegenerative disease. Regenerative Medicine Nov; 5(6):933-46, 2010.
Kambal A, Mitchell G, Cary W, Gruenloh W, Jung Y, Kalomoiris S, Nacey C, McGee J, Lindsey M, Fury B, Bauer
G, Nolta JA, Anderson JS. Generation of HIV-1 Resistant and Functional Macrophages From Hematopoietic Stem
Cell-derived Induced Pluripotent Stem Cells. Molecular Therapy Mar; 19(3):584-93, 2011.
Chen X, Lingala S, Zern M, Nolta J., Wu J. Epithelial mesenchymal transition and hedgehog signaling activation are
associated with chemoresistance and invasion of hepatoma subpopulations. J. Hepatology;55(4):838-45, 2011.
Meyerrose T, Olson S, Pontow S, Kalomoiris S, Jung Y, Annett G, Bauer G, Nolta JA. Mesenchymal stem cells for
the sustained in vivo delivery of bioactive factors. Advances in Drug Delivery Reviews 62 (12) 1167-74, 2010.
Gruenloh W, Kambal A, Sondergaard C, McGee J, Nacey C, Olson S, Fierro F, Nolta JA, Characterization and In
Vivo Testing of Mesenchymal Stem Cells Derived From Human Embryonic Stem Cells. Tissue Engineering 17 (11)
1517-25, 2011.
Fierro FA, Kalomoiris S, Nolta JA. Effects on proliferation and differentiation of multipotent mesenchymal stromal
cells over-expressing growth factors for combined gene/cell therapy. Stem Cells 29(11):1727-37, 2011.
Jun-feng Feng, Jing Liu, Xiu-zhen Zhang, Lei Zhang, Ji-yao Jiang, Jan Nolta, Min Zhao. Guided Migration of Neural
Stem Cells Derived from Human Embryonic Stem Cells by an Electric Field. Stem Cells. Feb; 30(2):349-55, 2012.
Y Jung, G Bauer, and J Nolta. Induced pluripotent stem cells and mesenchymal stem cells: progress toward safe
clinical products. Stem Cells. Jan;30(1):42-7, 2012.
Murphy WJ, Nolta JA. Autoimmune T Cells Lured to a FASL Web of Death by MSCs. Cell Stem Cell. May
4;10(5):485-7, 2012.
85. Kumari R, Li H, Haudenschild DR, Fierro F, Carlson CS, Overn P, Gupta L, Gupta K, Nolta J, Yik JH, Di Cesare
PE. The oncogene LRF is a survival factor in chondrosarcoma and contributes to tumor malignancy and drug
resistance. Carcinogenesis. Nov;33(11):2076-83, 2012.
86. Mathews G, Sondergaard C, Jeffreys A, Childs W, Le B, Sahota A, Najibi S, Nolta J, Si MS. Computational Analysis
of Contractility in Engineered Heart Tissue. IEEE Trans Biomed Eng. May; 59(5):1429-35, 2012.
87. Walker JE, Chen RX, McGee J, Nacey C, Pollard RB, Abedi M, Bauer G, Nolta JA, Anderson JS. Generation of an
HIV-1-resistant immune system with CD34(+) hematopoietic stem cells transduced with a triple-combination antiHIV lentiviral vector. J Virol. May; 86(10):5719-29, 2012.
88. Park SS, Caballero S, Bauer G, Shibata B, Roth A, Fitzgerald PG, Forward KI, Zhou P, McGee J, Telander DG, Grant
MB, Nolta JA. Long-Term Effects of Intravitreal Injection of GMP-Grade Bone-Marrow-Derived CD34+ Cells in
NOD-SCID Mice with Acute Ischemia-Reperfusion Injury. Invest Ophthalmol Vis Sci.; 53(2):986-94, 2012.
89. S Olson, K Pollock, A Kambal, W Cary, G Mitchell, J Tempkin, H Stewart, J McGee, G Bauer, T Tempkin, V
Wheelock, G Annett, G Dunbar and J Nolta, Genetically Engineered Mesenchymal Stem Cells as a Proposed
Therapeutic for Huntington’s disease. Molecular Neurobiology;45(1):87-98, 2012.
90. Kalomoiris S, Lawson J, Chen RX, Bauer G, Nolta JA, Anderson JS. CD25 Preselective Anti-HIV Vectors for
Improved HIV Gene Therapy. Hum Gene Ther Methods. 2012 Dec; 23(6):366-75.
91. S Olson, A Kambal, K Pollock, G Mitchell, H Stewart, S Kalomoiris, W Cary, C Nacey, K Pepper, J Nolta.
Examination of mesenchymal stem cell-mediated RNAi transfer to Huntington's disease affected neuronal cells for
reduction of huntingtin. Molecular and Cellular Neuroscience;49(3):271-81, 2012.
92. Liu J, Githinji J, McLaughlin B, Wilczek K, Nolta J. Role of miRNAs in Neuronal Differentiation from Human
Embryonic Stem Cell-Derived Neural Stem Cells. Stem Cell Rev;8(4):1129-37, 2012.
93. Liu J, Koscielska KA, Cao Z, Hulsizer S, Grace N, Mitchell G, Nacey C, Githinji J, McGee J, Garcia-Arocena D,
Hagerman RJ, Nolta J, Pessah I, Hagerman PJ. Signaling defects in iPSC-derived fragile X premutation neurons.
Hum Mol Genet. 21(17):3795-805. 2012.
94. M Guan, R Liu, K Lam, J Lou, Nolta J, B Panganiban, R Ritchie, N Lane, W Yao. Directing mesenchymal stem cells
to bone to increase bone formation. Nature Medicine; 18(3):456-62, 2012.
95. Annett G, Bauer G, Nolta JA. Mesenchymal stem cells for trinucleotide repeat disorders. Methods Mol Biol.
1010:79-91, 2013.
96. Ma X, Duan Y, Tschudy-Seney B, Roll G, Behbahan IS, Ahuja TP, Tolstikov V, Wang C, McGee J, Khoobyari S,
Nolta JA, Willenbring H, Zern MA. Highly Efficient Differentiation of Functional Hepatocytes From Human
Induced Pluripotent Stem Cells. Stem Cells Transl Med. May 16. [Epub ahead of print] 2013.
97. NL Magner, Y Jung, J Wu, JA Nolta, MA Zern, and P Zhou. Insulin and IGFs Enhance Hepatocyte Differentiation
from Human Embryonic Stem Cells via the PI3K/AKT Pathway. Stem Cells. 2013 Oct;31(10):2095-103.
98. Plowman EK, Bijangi-Vishehsaraei K, Halum S, Cates D, Hanenberg H, Domer AS, Nolta JA, Belafsky PC.
Autologous myoblasts attenuate atrophy and improve tongue force in a denervated tongue model: A pilot study.
Laryngoscope. 2013 Aug 8. 124(2): 20-6. PMID:23929623
99. Yao W, Guan M, Jia J, Dai W, Lay YA, Amugongo S, Liu R, Olivos D, Saunders M, Lam K, Nolta J, Olvera D,
Ritchie RO, Lane NE. Reversing Bone Loss by Directing Mesenchymal Stem Cells to the Bone. Stem Cells. 2013 Jul
2. doi: 10.1002/stem.1461. [Epub ahead of print].
100. Binder BY, Sondergaard CS, Nolta JA, Leach JK. Lysophosphatidic Acid enhances stromal cell-directed
angiogenesis. PLoS One. 2013 Dec 2;8(12):e82134. doi: 10.1371/journal.pone.0082134
101. Gericota B, Anderson JS, Mitchell G, Borjesson DL, Sturges BK, Nolta JA, Sieber-Blum M. Canine Epidermal
Neural Crest Stem Cells: Characterization and Potential as Therapy Candidate for a Large Animal Model of Spinal
Cord Injury. Stem Cells Transl Med. 2014 Jan 17. [Epub ahead of print]
102. Dasu MR, Ramirez SR, La TD, Gorouhi F, Nguyen C, Lin BR, Mashburn C, Stewart H, Peavy TR, Nolta JA, Isseroff
RR. Crosstalk Between Adrenergic and Toll-Like Receptors in Human Mesenchymal Stem Cells and Keratinocytes:
A Recipe for Impaired Wound Healing. Stem Cells Transl Med. 2014 Jun;3(6):745-59. doi: 10.5966/sctm.2013-0200.
Epub 2014 Apr 23. PMID: 24760207
103. Perez-Cunningham J, Ames E, Smith RC, Peter AK, Naidu R, Nolta JA, Murphy WJ. Natural killer cell subsets
differentially reject embryonic stem cells based on licensing. Transplantation. 2014 May 27;97(10):992-8. PMID:
24704665
104. Clark EA, Kalomoiris S, Nolta JA, Fierro FA. Concise review: MicroRNA function in multipotent mesenchymal
stromal cells. Stem Cells. 2014 May;32(5):1074-82. Review. PMID: 24860868
105. Osborn SL, Thangappan R, Luria A, Lee JH, Nolta J, Kurzrock EA. Induction of human embryonic and induced
pluripotent stem cells into urothelium. Stem Cells Transl Med. 2014 May;3(5):610-9.
106. McMahill BG, Borjesson DL, Sieber-Blum M, Nolta JA, Sturges BK. Stem Cells in Canine Spinal Cord Injury Promise for Regenerative Therapy in a Large Animal Model of Human Disease.Stem Cell Rev. 2014 Aug 31. [Epub
ahead of print] PMID: 25173879
107. Satake N, Duong C, Chen C, Barisone GA, Diaz E, Tuscano J, Rocke DM, Nolta J, Nitin N. Targeted therapy with
MXD3 siRNA, anti-CD22 antibody and nanoparticles for precursor B-cell acute lymphoblastic leukaemia. Br J
Haematol. 2014 Nov;167(4):487-99. doi: 10.1111/bjh.13066. Epub 2014 Sep 8. PMID: 25196579
108. Gao W, Zhou P, Ma X, Tschudy-Seney B, Chen J, Magner NL, Revzin A, Nolta JA, Zern MA, Duan Y. Ethanol
Negatively Regulates Hepatic Differentiation of hESC by Inhibition of the MAPK/ERK Signaling Pathway In Vitro.
PLoS One. 2014 Nov 13;9(11):e112698. doi: 10.1371/journal.pone.0112698. eCollection 2014. PMID: 25393427
109. Nolta JA. Editorial: 2014 --New advances in understanding Stem Cell fate and function. Stem Cells. 2014 Nov 28.
doi: 10.1002/stem.1905. [Epub ahead of print] PMID:25446041
110. Park SS, Bauer G, Abedi M, Pontow S, Panorgias A, Jonnal RS, Zawadzki RJ, Werner JS, Nolta J. Intravitreal
Autologous Bone Marrow CD34+ Cell Therapy For Ischemic and Degenerative Retinal Disorders: Preliminary Phase
1 Clinical Trial Findings. Invest Ophthalmol Vis Sci. 2014 Dec 9. pii: IOVS-14-15415. doi: 10.1167/iovs.14-15415.
[Epub ahead of print] PMID: 25491299
111. Osborn SL, So M, Hambro S, Nolta JA, Kurzrock EA. Inosculation of blood vessels allows early perfusion and
vitality of bladder grafts-implications for bioengineered bladder wall. Tissue Eng Part A. 2015 Jun;21(11-12):190615. doi: 10.1089/ten.TEA.2014.0630. Epub 2015 Apr 22.
112. Barclay SL, Yang Y, Zhang S, Fong R, Barraza A, Nolta JA, Torbett BE, Abedi M, Bauer G, Anderson JS. Safety
and efficacy of a tCD25 pre-selective combination anti-HIV lentiviral vector in human hematopoietic stem and
progenitor cells. Stem Cells. 2015 Mar;33(3):870-9. PMID: 25524029
113. Brian Fury, Tia Hackett, Fernando Fierro, Jan Nolta, Gerhard Bauer. Clinically relevant formulation and conditions
for transportation of genetically modified bone marrow mesenchymal stem cells engineered to overexpress vascular
endothelial growth factor. Cytotherapy 06/2015; 17(6):S42. DOI:10.1016/j.jcyt.2015.03.453 ·
114. Black Amanda B., Dahlenburg Heather, Pepper Karen, Nacey Catherine, Pontow Suzanne, Kuhn Maggie A.,
Belafsky Peter C., and Nolta Jan A. Human Myoblast and Mesenchymal Stem Cell Interactions Visualized by
Videomicroscopy. Human Gene Therapy Methods. Online ahead of print. doi:10.1089/hgtb. 2015.100. 2015.
115. Beegle J, Lakatos K, Kalomoiris S, Stewart H, Isseroff RR, Nolta JA. Hypoxic Preconditioning of Mesenchymal
Stromal Cells Induces Metabolic Changes, Enhances Survival and Promotes Cell Retention in Vivo. Stem Cells. 2015
Feb 19;PubMed PMID: 25702874.
116. Kol A, Arzi B, Athanasiou KA, Farmer DL, Nolta JA, Rebhun RB, Chen X, Griffiths LG, Verstraete FJ, Murphy CJ,
Borjesson DL. Companion animals: Translational scientist's new best friends. Sci Transl Med. 2015 Oct
7;7(308):308ps21. doi: 10.1126/scitranslmed.aaa9116. Review. PMID:26446953
Books, Invited Articles, and Chapters (Selected, of over 50)
1.
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4.
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9.
Dao MA and Nolta JA. Retroviral - mediated transduction of human hematopoietic stem and progenitor cells
measured in a long-term xenograft assay. European Society of Hematology Teaching Manual, Plenum Press, 1997.
Nolta JA, and Kohn DB. Human hematopoietic cell culture, transduction, and analyses. “Current Protocols in Human
Genetics”, John Wiley and Sons, Inc. Chapter 13, Unit 13.7, 1997.
Dao MA and Nolta JA. The effects of ex-vivo manipulation on the differentiation of human hematopoietic stem cells.
Teaching Manual for the Annual Meeting of the American Association of Blood Banks, AABB Press, Chapter 4,
12110-12135, 1998.
Kohn DB, Nolta JA, Crooks GM. Clinical trials of gene therapy using hematopoietic stem cells. Hematopoietic Cell
Transplantation, 2nd edition. Edited by Forman S, Blume K, Thomas ED. Blackwell Scientific Publications, Boston,
MA. Chapter 11; Pages 97-102, 1999.
Kohn DB, Scheuning F, Dunbar C, Pensiero M, Kiem HP, Beach K, Zaboikin M, Miller D, Storb R, Wells S, Nolta
J, Esplin J, Barton N, Brady R, and Karlsson S. Early trials of gene transfer for Gaucher Disease. Advances in
Jewish Genetic Diseases, 2001.
Kohn DB, Crooks GM, Nolta JA. Gene therapy using hematopoietic stem cells. Hematopoietic stem cell therapy,
Ed.s: AD Ho and RE Champlin. Cambridge University Press, 2001.
Dao MA and Nolta JA. An increase in the levels of retroviral - mediated transduction of engrafting human
hematopoietic progenitors can be obtained by manipulation of the hematopoietic cell cycle. Molecular Biology of
Hematopoiesis VI, Edited by Abraham et al., Plenum Publishers, NY 2001.
Kohn DB, Crooks GM, Nolta JA, Kapoor N, Weinberg K, and Parkman, R. Gene therapy with cord blood
hematopoietic stem cells. Immunologic function of cord blood hematopoietic stem cells. Ed Paul McCurdy, M.D.,
Marcel Dekker Inc, New York. 2001.
Dao MA, Nolta JA. Retroviral - mediated transduction of human hematopoietic stem and progenitor cells.
Hematopoietic Stem Cell Protocols, Chapter 17, P. 253-274. Editors. C. Jordan and C. Klug. The Humana Press Inc.,
Totowa, NJ 2002.
10. Meyerrose TM, Herrbrich P, Wang X, Dao MA, Nolta JA. Pluripotent human stem cell analysis in immune deficient
mice. The Pluripotential Hematopoietic Stem Cell, Editor: J. Keller. Eurekah Press, Chapter 4, 2003.
11. Meyerrose TE, Herrbrich P, Hess DA, Nolta JA. Immune-deficient mouse models for analysis of human stem cells.
Biotechniques. Dec; 35 (6):1262-72, 2003.
12. Nolta J, Bauer G, and Sands M. Animal models for clinical gene therapy trials. In Translational and Experimental
13.
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Clinical Research. Philadelphia: Lippincott Williams & Wilkins; Schuster, Daniel P. and William J. Powers, eds.
2005.
Nolta JA and Kohn DB. Hematopoietic stem cells for gene therapy. In Stem Cell Handbook, Edited by C. S. Potten,
Academic Press Limited, London, UK. Pages 447-461, 1996.
Meyerrose TM, Herrbrich P, Wang X, Dao MA, Nolta JA. Pluripotent human stem cell analysis in immune deficient
mice. The Pluripotential Hematopoietic Stem Cell, Editor: J. Keller. Eurekah Press, 2003.
Meyerrose TE, Herrbrich P, Hess DA, Nolta JA. Immune-deficient mouse models for analysis of human stem cells.
Biotechniques. Dec; 35 (6):1262-72, 2003.
Meyerrose T, Rosova I, Dao M, Bauer G, and Nolta JA. Establishment and transduction of primary human
mesenchymal stem cell monolayers. Chapter 2 in Genetic Engineering of Mesenchymal Stem Cells. Kluwer, pp 4558. Nolta JA (Editor). Feb 2006.
Jesper Bonde, Donald B. Kohn, and Jan A. Nolta. Human Hematopoietic Cell Culture, Transduction, and Analyses.
Current Protocols in Human Genetics. Chapter 13, 2008.
Ping Zhou, Sarah Hohm, Ben Capoccia, Dan Link, and Nolta JA. Immunodeficient mouse models to study human
stem-cell mediated repair of tissue injury. Hematopoietic Stem Cell Protocols: Second Edition, Methods in Molecular
Medicine, The Humana Press Inc., edited by Kevin Bunting. 2008.
FA. Fierro, J Egana, C Toupadakis, C Yellowley, H Machens, JA Nolta. Small Animal models of tissue regeneration.
Chapter 20 in Stem Cell Biology and Regenerative Medicine. H. Bernstein (Editor), 2011.
Armstrong L, Lako M, Buckley N, Lappin TR, Murphy MJ, Nolta JA, Pittenger M, Stojkovic M. Our top 10
developments in stem cell biology over the last 30 years. Stem Cells. Jan; 30(1):2-9, 2012.
Y Jung, G Bauer, and J Nolta. Induced pluripotent stem cells and mesenchymal stem cells: progress toward safe
clinical products. Stem Cells. Jan; 30(1):42-7, 2012.
G Annett, G Bauer and J Nolta. Mesenchymal Stem Cells for Trinucleotide Repeat Disorders. Chapter 12 in
Trinucleotide Repeat Protocols, Second Edition, edited by Kohwi and McMurray, 2013.
Nolta JA. Editorial: 2013 - a year of clinical success and great scientific innovation in the stem cell field. Stem Cells.
2014 Jan; 32(1):1-2. PMID:24375745.
Lee JK, Responte DJ, Cissell DD, Hu JC, Nolta JA, Athanasiou KA. Clinical translation of stem cells: insight for
cartilage therapies. Crit Rev Biotechnol. 2014 Mar; 34(1):89-100. PMID: 24083452
Hagerman R, Moghaddam B, Nolta J, Diez-Juan M, Hendren R. Treatment of Neurodevelopmental Disorders: Targeting
Neurobiological Mechanisms. Chapter in: Translating Treatments from the Laboratory to the Clinic. Oxford
University Press, 2014Concise review: MicroRNA function in multipotent mesenchymal stromal cells. Clark EA,
Kalomoiris S, Nolta JA, Fierro FA. Stem Cells. 2014 May;32(5):1074-82. PMID: 24860868
Relevant Investigator-initiated INDs/Clinical Trials (blue-NIH-RAC numbers for gene therapy trials, Nolta subinvestigator):
0301-566 Infusion of Genetically Modified T cells: Tracking and Toxicity (DiPersio)
0602-758 Lentiviral-Mediated Gene Therapy for MPS VII (Sands)
9306-047 Retroviral Mediated Transfer of the cDNA for Human Glucocerebrosidase into Stem Cells of Patients with
Gaucher Disease. (Kohn)
NCT00794508: Transduction of CD34+ Cells from Children with Adenosine Deaminase (ADA)-Deficient SCID (Kohn)
NCT00871702: Infusion of Genetically Modified T Cells for Post-Transplant Patients with Relapsed Disease (DiPersio)
FDA IND: BB IND# 13065. Stem Cell Implantation in Elderly Patients Undergoing CABG (Rich)
FDA IND: BB IND# 14848. Infusion of autologous cord blood for traumatic brain injury
FDA IND: BB IND# 13307. A Pilot Clinical Trial of the Feasibility and Safety of Intravitreal Autologous Adult Bone Marrow
Stem Cells in Treating Eyes With Vision Loss From Retinopathy (Park).
FDA IND: BB IND# 16129. "Autologous Peripheral Blood CD34+ (Miltenyi CliniMACS) cells Transduced with lentiviral Vector
(ITAX) Expressing Chimeric Human/Rhesus TRlM5-alpha, CCR5 shRNA, a TAR decoy, and Truncated Human tCD25 genes;
Cultured with Cytokines; Following Chemotherapy.
Patent Applications/Patents
- Mesenchymal Stem Cells producing inhibitory RNA for disease modification. ROI 2008. UC Patent 2009-170-1; Ref.:
060933-4300. Inventors: Scott Olson, Ph.D., Louisa Wirthlin, B.S., Jan Nolta Ph.D. International Filing 2009.
- Combination anti-HIV vectors, targeting vectors, and methods of use. PCT/US2010/033042. Inventors J. Anderson, G.
Bauer, Jan Nolta, 2009.
- EIIIA and EIIIB segments of fibronectin regulate stem cell fate. 060933-5650. S.Pontow, J. Peters, Jan Nolta. 2010.
- HIV resistant immune system cells from induced pluripotent stem cells. J. Anderson, G. Bauer, Jan Nolta, ROI 2010
- Decellularized liver matrix and iPSC-derived hepatocytes for liver bioengineering. J. Wu, P. Zhou, J Nolta, ROI 2010.
- Genetically modified MSC and therapeutic methods. Fernando Fierro, Karen Pepper, Gerhard Bauer and Jan Nolta, PCT
International application no. PCT/US2013/057721, filed on August 30, 2013.
-Vectors and Methods to Treat Ischemia. Fernando Fierro, Karen Pepper, Gerhard Bauer and Jan Nolta, PCT
International application no. PCT/US2014/029386, filed on March 14, 2014
Jan A. Nolta, Ph.D. - Research Projects - Active:
1) 1R01GM099688-01 (Nolta) 07/01/2011 - 06/30/2016
1.8 months effort
NIH Common fund transformative research projects program
Direct cell to cell transfer of microRNA for tissue repair. The studies will examine the use of human mesenchymal
stem cells to deliver microRNA and other key factors to damaged tissues to hasten repair.
No overlap
2) CIRM DR2A-05415 (Wheelock PI/Nolta Co-PI)
11/01/12-10/31/16
1.8 mo effort
MSC engineered to produce BDNF for the treatment of Huntington's disease. This award is to prepare and conduct a
Phase I clinical trial of human Mesenchymal Stem Cells engineered to express brain – derived neurotrophic factor for
patients with early stage Huntington’s disease. It is based on preclinical data demonstrating the safety and efficacy of this
approach in rodent and non-human primate models. The goal is to restore striatal brain volume and to reduce levels of
movement and cognitive decline in HD patients.
No overlap
3) CIRM DR2A-05423 (Laird PI/Nolta co-PI)
11/01/12 – 10/31/16 1.8 mo effort
Phase I study of IM Injection of VEGF Producing MSC for the Treatment of Critical Limb Ischemia. This award is
to prepare and conduct a Phase I clinical trial of human Mesenchymal Stem Cells engineered to express VEGF for
patients with end-stage critical limb ischemia. The goal is to prevent amputation of limbs.
No overlap
4) TR2-01787: CIRM (Isseroff PI, Nolta Co-PI)
02/01/11-01/31/14
1.8 months (15%)
Bone Marrow Mesenchymal Stem Cells to Heal Chronic Diabetic Wounds
The goal of the proposed studies is to move a development candidate composite stem cell/ decellularized matrix construct
to the clinic for non-healing ulcers in diabetic patients seen in Dr. Isseroff’s wound care clinic at the VA hospital.
No Overlap
5) RB4-05764 (PI-Lieu, Co-Investigator/mentor Nolta)
California Institute for Regenerative Medicine 2/1/13 – 1/31/16
0.6 cal mos.
Induction of pluripotent stem cell-derived pacemaking cells. This proposal studies the mechanisms of cardiac cell
differentiation from human induced pluripotent stem cells to generate biopacemakers.
No Overlap
6) TB1-01184: CIRM (Trainer, Jill PI, Nolta UC Davis lead mentor) 10/01/09 – 09/31/14
0.48 months (4%)
Bridges to Stem Cell Research Training Program. Bridges is an innovative collaborative program between California
State University Sacramento (CSUS) and UC Davis to train state college Master’s Program interns in the practical aspects
of stem cell biology, good manufacturing practices, good laboratory practices, and Cellular therapy. Subcontract
No overlap
Previous Grants
7) CIRM FA-00611-1: Institutional grant: Nolta, designated contact,
CIRM Major Facilities Grant: this award was for “Bricks and Mortar” and equipment for renovation of the new UC
Davis/CIRM Institute for Regenerative Cures, which is now the hub for the large UC Davis stem cell program (currently
over 150 faculty members).
8) TR1-01257: CIRM (Nolta)
08/01/09-10/31/12
Sustained siRNA production from human MSC to treat Huntington’s disease and other neurodegenerative
disorders. Develop a novel MSC-based stem cell therapy to treat Huntington's disease, leading toward clinical trials. This
work is continuing and currently funded by the NIH Office of the Director.
9) 2RO1DK61848 (Nolta), NIH NIDDK
09/30/01 – 06/30/11
Immunodeficient mouse model of stem cell plasticity. The goal of the proposed studies was to develop immune
deficient mouse models to track the origin of blood and liver cells, to determine whether a common progenitor existed. A
common stem cell precursor could have been validated by the single cell clonal integration analysis techniques developed
in the Nolta lab. The work resulted in several reports that, through careful clonal marking studies, the adult blood and
liver stem cells were in fact demonstrated to be distinct.
10) RN1-00561: CIRM (Chong Pan PI, Nolta Co-mentor)
2008-2013
0.6 months (5%)
Combinatorial Chemistry Approaches to Develop Ligands to Leukemia Stem Cells. Dr. Nolta was Dr. Chong’s stem
cell mentor. This work resulted in several potential drugs, now in the patent process.
11) RM1-01724: CIRM (Murphy PI, Nolta co-investigator) 2010-2013
1.2 months (10%) effort
Natural killer (NK) cells as “veto” cells to promote donor-specific tolerance. Develop methods to tolerize recipients
to engrafted tissues by the use of donor NK cells. This work is ongoing and leading toward clinical trials at UCD with an
industry partner.
12) TR2-01857: CIRM (Zern PI, Nolta Co-Investigator) 2011-2013 1.2 months (10%) effort
Liver Cell Transplantation. Establish human ESCs as a source of hepatocytes for the future planned therapy of patients
with liver failure. This work is now being continued by Co-PI Dr. Willenbring at UCSF.
13) 1RO1 HL073256-01 (Nolta) NIH NHLBI 07/01/03 - 06/30/12
1.8 months (15%) Effort
Functional Dissection of human adipose-derived MSC and bone marrow-derived MSC phenotypes. The goal was
to identify the most primitive subsets of primary human MSC and to determine the functional differentiative potential of
each defined subset and the homing and tissue regenerative potential in immune deficient mice. The standard operating
procedures for human MSC expansion and transduction resulting from this funding are now used in the CMC section for
multiple IND applications at UC Davis. These SOPs form the basis of the cellular product for the CIRM disease team
grant and proposed trial “Phase I study of IM Injection of VEGF Producing MSC for the Treatment of Critical Limb
Ischemia.”
14) 2P51RR000169-49. Stem Cell Therapies for Huntington’s disease 2011-2012
To conduct IND-enabling studies with the UC Davis Clinical Translational Science Center (CTSC) in support of a stem
cell clinical trial for HD. Non-human primate biosafety studies were conducted. These data provided preliminary
evidence of safety for CIRM grant DR2A-05415 “MSC engineered to produce BDNF for the treatment of Huntington's
disease”.
15) RB2-01567: CIRM (Kurzrock PI, Nolta co-PI)
2010-2013
0.6 months (5%)
Differentiation of Human Stem Cells into Urothelium. The goal is to develop bladder tissue replacement constructs for
patients with cancer and spin bifida, both causing loss or absence of functional bladder tissue. This project is ongoing and
currently funded by Shriner’s Hospital of Northern California.
16) 5RC1AG036022-02: ARRA Challenge grant (Hagerman PI, Nolta, Pesseh co-Is) 10/2009 -09/2011
Human iPSC neuronal models for FXTAS neurodegeneration. Generate induced pluripotent stem cells from the
fibroblast cell lines from the consented FXTAS patients seen by the Hagermans and to study the potential signaling
deficits from neurons derived from each pluripotent cell line, in comparison to unaffected controls.
17) 1S10RR026825-01 (Nolta) NIH NCRR
01/26/10
Shared LSRII Cytometer Equipment Application – this award funded a shared cytometer for the Stem Cell Program’s
Sorter Core in the new Institute for Regenerative Cures. The instrument is used heavily in the Institute’s Flow core.
18) 5P30AG010129 - 19 (DeCarli, PI, Nolta - Pilot grant PI)
10/09-09/10
The goal of this pilot project on Dr. DeCarli’s center grant was for Dr. Nolta to mentor Dr. Olson in her lab, who will
investigate the use of human MSC to deliver biotherapeutics for Alzheimer’s disease directly into the brain tissue.
19) 1R01HL073762-01 (Link), NIH NHLBI
07/01/03 – 06/30/07
Stem Cell Mobilization to enhance tissue repair. Regimens to mobilize murine hematopoietic, mesenchymal, and
endothelial progenitors capable of mediating repair of damaged cardiac tissue were determined.
20) 2R01DK53041 (Nolta), NIH NIDDK
09/30/97 - 04/30/07
Ex-vivo Stem Cell Manipulation and Human Hematopoiesis. Promote primitive human hematopoietic progenitor
survival and self-renewal in vitro, determine molecular changes that precede lineage commitment in human stem cells.
21) HL54850-02 (Nolta), NIH NHLBI
1/1/01 - 12/31/06
Specialized Center of Research (Parkman), Project 2 (Nolta); In vivo model for human stem cells. Examine
transduction, in vivo differentiation, and the molecular aspects of cell cycle regulation in human hematopoietic stem cells.
The results of this study, and SOPs developed, were used in several clinical trials of gene-modified hematopoietic stem
cells done at Children’s Hospital Los Angeles, to treat children with Primary Immune Deficiencies, including
“NCT00794508 Transduction of CD34+ Cells from Children with Adenosine Deaminase (ADA)-Deficient SCID
(Kohn)”.
22) 1R21DK62892-01 (Nolta) NIH NIDDK
03/01/03 – 02/28/06
HGF (Hepatocyte growth factor) induces robust human thymopoiesis in mice
The goal was to produce better xenograft models in which to humanize mice, to examine human T cell reactivity. The
models were modified by the UC Davis HIV team researchers, who have recently had an IND approved by FDA for their
pending clinical trial, “Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-selected CD34+ Cells
(Abedi and Anderson).”