BIOGRAPHICAL SKETCH Provide the following information for the Senior/key personnel and other significant contributors. Follow this format for each person. DO NOT EXCEED FOUR PAGES. NAME Claes Wahlestedt eRA COMMONS USER NAME (credential, e.g., agency login) clawah POSITION TITLE Leonard M. Miller Professor (Pending), University of Miami Miller School of Medicine EDUCATION/TRAINING (Begin with baccalaureate or other initial professional education, such as nursing, include postdoctoral training and residency training if applicable.) DEGREE INSTITUTION AND LOCATION MM/YY FIELD OF STUDY (if applicable) University of Lund, Sweden M.D. 01/86 Medicine University of Lund, Sweden Ph.D. 05/87 Pharmacology Kyoto University, Institute for Immunology, Kyoto, Post-Doctoral #1 06/88 Biotechnology Japan Georgetown University, Fidia-Georgetown Institute Neuropharmacology Post-Doctoral #2 05/89 for the Neurosciences, Washington D.C., USA & Molecular Biology Please refer to the application instructions in order to complete sections A, B, C, and D of the Biographical Sketch. A. Personal Statement I am a pharmacologist, molecular biologist, genome researcher and neuroscientist. In addition to my academic career, I have held several leadership positions in the pharmaceutical industry and I have been involved with smaller biotechnology companies. B. Positions Positions and Employment 1989-1993 Assistant Professor, Department of Neurology and Neuroscience, Cornell University Medical College, New York, NY, USA (Prof. D. J. Reis, Prof. F. Plum) 1993-1997 Founding Director and Head, Astra-Zeneca Research Centre Montreal, (~150 scientists) Montreal, Canada 1994-1997 Adjunct Professor of Pharmacology and Therapeutics, McGill University, Montreal 1994-1997 Adjunct Professor of Biochemistry, McGill University, Montreal 1997-2004 Director, Genomics and Biotechnology, Global R&D; served on various Leadership Teams, Pharmacia & Upjohn Inc./Pharmacia Corp./Pfizer Inc., Stockholm, Sweden/Peapack, NJ, USA 1997-2005 Founding Director, Department Chair and Professor, Center for Genomics and Bioinformatics (~200 Scientists), Department of Cell and Molecular Biology, Karolinska Institute, Stockholm, Sweden 2004-2010 Adjunct Chief Scientist, RIKEN Genomic Sciences Center, Yokohama and Wako/Tokyo, Japan 2005-2011 Professor and Director of Neuroscience Discovery, Departments of Neuroscience & Molecular Therapeutics, The Scripps Research Institute, Jupiter, FL, USA 2011Leonard M. Miller Professor (Pending); Associate Dean for Therapeutic Innovations; Vice Chair for Research, Department of Psychiatry and Behavioral Sciences; Center Director, Hussman Institute of Human Genomics, University of Miami Miller School of Medicine, Miami, FL, USA Selected Other Recent Experience 1997- Present Core contributor to the international Fantom Mammalian Genomics/Transcriptomics Consortium, Japan, Sweden, USA. 2000-2006 Chair, Nobel Conference Committees (Stockholm) 2000- Present Selected scientific advisory assignments for National programs: Genome Canada (Canada); Functional Genomics Program, FUGE (Norway); Genoma Espana (Spain); Academy of Finland (Drug 2000 Program Chair); Netherlands Organization for Scientific Research, NWO (The 2006-Present 2006-2010 2008–Present 2009–Present 2006-Present 2008 2010–Present 2011 Netherlands); Netherlands Genomics Initiative, NGI (The Netherlands); Department of Biotechnology (India); NIH Study Sections (USA): ZMH1 Special Emphasis Panel, 2009, 2010; ETTN, 2009- Present; BGES, 2007-2010; MNG 2011-Present. Advisory Board of Florida Center for Brain Tumor Research Board of Directors, Florida Blood Centers, Orlando, FL Board of Directors, St. Mary’s Medical Center, West Palm Beach, FL Director and Program Chair (2009, 2011), Nature Publishing Group / University of Miami / Scripps; Miami Winter Symposia Chair, Florida Center for Universal Research to Eradicate Disease (FLCURED) (Florida Department of Health), Tallahassee, FL) Co-founded CURNA Inc. (acquired by OPKO Health in 2011) Appointed by Governor Charlie Crist to the Biomedical Research Advisory Council of Florida Carnegie Centenary Professorship Award, Carnegie Trust for the Universities of Scotland, Edinburgh, UK C. Fifteen relevant peer-reviewed publications (from 193 of which 29 have been cited >100 times for a total of >14,000 ISI citations). 1. Hollander JA, Im H, Amelio A, Kocerha J, Bali P, Lu Q, Willoughby D, Wahlestedt C, Conkright M, Kenny PJ. Striatal microRNA controls cocaine intake through CREB signaling. Nature 466:197-202, 2010. 2. Faghihi M.A. and Wahlestedt C. Regulatory roles of natural antisense transcripts. Nature Reviews Molecular Cell Biology 10(9):637-43, 2009. 3. FANTOM Consortium. The transcriptional network that controls growth arrest and differentiation in a human myeloid leukemia cell line. Nature Genetics 41(5):553-62, 2009. 4. Kocerha J, Faghihi MA, Lopez-Toledano MA, Huang J, Ramsey AJ, Caron MC, Sales N, Willoughby DA, Elmen J, Hansen HF, Orum H, Kauppinen S, Kenny PJ and Wahlestedt C. MicroRNA-219 modulates NMDA receptor mediated neurobehavioral dysfunction. Proc Natl Acad Sci (USA) 106(9):3507-12, 2009. 5. Faghihi MA, Modarresi F, Khalil AM, Wood DE, Sahagan BG, Morgan TE Finch CE, Kenny PJ and Wahlestedt C. Expression of a noncoding RNA is elevated in Alzheimer’s disease and drives rapid feed-forward regulation of beta-secretase. Nature Medicine 14(7):723-30, 2008. 6. Shan G, Li Y, Zhang J, Li W, Szulwach KE, Duan R, Faghihi MA, Khalil AM, Lu L, Paroo Z, Chan AW, Shi Z, Liu Q, Wahlestedt C, He C, Jin P. A small molecule enhances RNA interference and promotes microRNA processing. Nature Biotechnology 26(8):933-40, 2008. 7. Wahlestedt C. Antisense and non-coding RNA transcripts as potential drug targets. Drug Disc Today 11, (11-12), 503-8, 2006. 8. Carninci P, Sandelin A, Lenhard B, Katayama S, Shimokawa K, Ponjavic J, Semple CA, Taylor MS, Engstrom PG, Frith MC, Forrest AR, Alkema WB, Tan SL, Plessy C, Kodzius R, Ravasi T, Kasukawa T, Fukuda S, Kanamori-Katayama M, Kitazume Y, Kawaji H, Kai C, Nakamura M, Konno H, Nakano K, Mottagui-Tabar S, Arner P, Chesi A, Gustincich S, Persichetti F,Suzuki H, Grimmond SM, Wells CA, Orlando V, Wahlestedt C, et al. Genome-wide analysis of mammalian promoter architecture and evolution. Nature Genetics 38, 626-635. 2006. 9. Katayama S, Tomary T, Kasukawa T, Waki K, Suzuki M, Nishida H, Kawai J, Suzuki H, Carninci P, Hayashizaki Y, Wells C, Frith M, Ravasi T, Pang K, Mattick J, Hume D, Lipovich L, Engstrom PG, Mizuno Y, Faghihi MA, Sandelin A, Chalk A, Mottagui-Tabar S, Lenhard B, Wahlestedt C. Antisense transcription in the mammalian transcriptome. Science 309:1564-1566, 2005. 10. Carninci P*, Wahlestedt C*, et al. The transcriptional landscape of the mammalian genome. Science 309: 1559-1563, 2005. (*Core contributors) 11. Elmén J, Thonberg H, Ljungberg K, Frieden M, Westergaard M, Xu Y, Wahren B, Liang Z, Ørum H., Koch T, Wahlestedt C. Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality. Nucleic Acids Res 33(1), 439-447, 2005. 12. Okazaki Y*, Wahlestedt C*, et al. Analysis of the mouse transcriptome based on functional annotation of 60,770 full-length cDNAs. Nature 420:563-573, 2002. (*Core contributors) 13. Wahlestedt C, Salmi P, Good L, Kela J, Johnsson T, Hökfelt T, Broberger C, Porreca F, Lai J, Ren K, Ossipov M, Koshkin A, Jakobsen N, Skouv J, Ørum H, Jacobsen MH, Wengel J. Potent and nontoxic antisense oligonucleotides containing locked nucleic acids. Proc Natl Acad Sci (USA) 97: 5633-5638, 2000. 14. Wahlestedt, C., Pich, E.M., Koob G.F., Yee, F. and Heilig, M.: Modulation of anxiety and neuropeptide Y-Y1 receptors by antisense oligodeoxynucleotides. Science, 259: 528-531, 1993. 15. Wahlestedt, C., Golanov, E., Yamamoto, S., Yee, F., Ericson, H., Yoo, H., Inturrisi, C.E. and Reis, D.J.: Antisense oligodeoxynucleotides to the NMDA-R1 receptor channel protect cortical neurones from excitotoxicity and reduce focal ischemic infarctions. Nature, 363: 260-263, 1993. D. Research Support (I relocated to the USA from Sweden in 2005 and it was not possible to transfer significant funding.) ONGOING NIH SUPPORT R01MH083733 (Wahlestedt/Kenny) Role of noncoding RNAs in Schizophrenia 07/01/08 - 02/28/13 NIH/NIMH R01DA025983 (Kenny/Wahlestedt) 09/15/08 - 06/30/13 Role of microRNAs in the Mechanisms of Drug Dependence NIH/NIDA R01NS063974 (Wahlestedt) 02/01/09 - 01/31/13 Regulatory RNAs as Mediators and Biomarkers in Alzheimer's Disease NIH/NINDS R01AA017943 (Wahlestedt) 06/01/09 - 05/31/11 NIH/NIAAA Discovery and Development of Nociceptin Receptor Ligands in Alcohol Dependence RC2AG03659 (Wahlestedt) 09/30/09 – 08/31/11 Noncoding RNAs As Epigenomic Modulators In Alzheimer's Disease U01 AA018665 (Wahlestedt) 04/10/10-03/31/14 Discovery of Potent and Selective Neuropeptide Y Y2 Receptor Antagonist Probes R01MH084880 (Wahlestedt) 04/26/10-01/31/15 Comprehensive Analysis of the FMR1 Locus Transcriptional Landscape COMPLETED NIH SUPPORT 1 R21 NS056950-01 (plus two supplements) 07/01/06 - 06/30/09 Assay Development Relating to the HTS of the Neuropeptide Y-Y2 Receptor 1 R21 AGO29290-02 07/01/07 - 04/30/09 RNAi strategy for prolonged inhibition of BACE1 NIH/NIA NIH/NIAAA NIH/NIMH NIH/NINDS NIH/NIA RL 1 AG032119-01 (with P. Hagerman) 09/30/07 – 06/30/10 NIH/NIA C1 consulting subcontract: Development of targeted therapeutic agents for the treatment of FXTAS