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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 POSITION TITLE Semple-Rowland, Susan L Professor of Neuroscience eRA COMMONS USER NAME (credential, e.g., agency login) Semple-Rowland 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) Gustavus Adolphus College, St. Peter, MN University of Pittsburgh, Pittsburgh, PA University of Florida, Gainesville, FL University of Florida, Gainesville, FL BA MSc PhD Post Doc 1977 1979 1986 1986-1989 Biology/Psychology Psychobiology Neuroscience Ophthalmology A. Personal Statement My research program focuses on development of lentiviral vector-based gene therapies for the treatment of Leber congential amaurosis – 1, an inherited retinal degeneration that is caused by null mutations in the gene encoding guanylate cyclase 1 (GC1). We have developed several different types of vector transgenes that we package into lentivirus and permit targeted delivery of multiple proteins to neural cells. In addition to my research program, my laboratory also designs, constructs, and packages vectors in collaboration with investigators interested in targeted delivery of genes to the CNS. B. Positions and Honors Positions and Employment 1979-1981 1990-1995 1995-1997 1998-2006 2006 – present 2000 – present 2003 –2010 Teaching Fellow, University of Pittsburgh, Pittsburgh, PA Assistant Research Scientist, University of Florida College of Medicine, Gainesville, FL Associate Research Professor, University of Florida College of Medicine, Gainesville, FL Associate Professor, University of Florida College of Medicine, Gainesville, FL Professor, University of Florida College Medicine, Dept. Neuroscience, Gainesville, FL Director, UF McKnight Brain Institute BSL-3 facility, University of Florida College of Medicine, Gainesville, FL Director IDP Neuroscience Graduate Program, University of Florida College of Medicine, Gainesville, FL Other Experience and Professional Memberships Grant reviewer 1998 1998-2000 2000 2001 2001 2001 2002 2003 – 2006 2004 2005 NIH/NEI VISB Study section/Special Emphasis panel Biochemistry/Molecular Biology Programming Committee for ARVO NIH/NEI VISB Study section/Special Emphasis panel NEI Board of Scientific Counselors, review Laboratory of Retinal Cell and Molecular Biology, NIH (Dec. 3-4) Ad hoc member of NINDS Training grant and Career Development Study section Reviewer for NSF Division of Sensory Biology and for the Division of Behavioral Neuroscience and NSF Behavioral Neuroscience NSF Sensory Systems and Computational Neuroscience Advisory Panel Fight-for-Sight Grant Review Board NEI special emphasis review panel Reviewer Special Emphasis NIH Panel – Gene Expression and Neuronal Development (11/9/05) Reviewer Vision SBIR BDCN IRG R43 grant (3/2/05) Reviewer for NSF Integrative Organismal Biology Section 3/20/06 Reviewer for Health Research Board – Ireland (12/07) Reviewer for NSF Neural Systems Activation Program Reviewer for Medical Research Council, London, England 2005 2006 2007 2008 2009 Editorial Boards 2001 – present Executive Editor for Experimental Eye Research 2004 – present Editorial Board Member – Molecular Vision Professional Societies 1986 – present Member Association for Research in Vision and Ophthalmology 2000 – present Member Society for Neuroscience Honors 1998 2000 2001 2003 2005 2006 2006-11 C. Lecture for Distinguished Scientist Seminar Series, University of Southern Alabama. Alcon Foundation Lecture in Ophthalmology - Emory University School of Medicine. Invited speaker Glaucoma Foundation 8th Annual Think Tank meeting on Gene Therapy University of Florida College of Medicine Exemplary Teacher Award UF College of Medicine Award for Outstanding Doctoral Dissertation Mentor Award Distinguished Alumni Citation in the field of Neuroscience –Gustavus Adolphus College, St. Peter, MN University of Florida College of Medicine Exemplary Teacher Award Selected Peer-reviewed Publications (Selected from 47 peer-reviewed publications) Most relevant to the current application Coleman JE, Huentelman MJ, Kasparov S, Metcalfe BL, Paton JF, Katovich MJ, Semple-Rowland SL, Raizada MK (2003) Efficient large-scale production and concentration of HIV-1-based lentiviral vectors for use in vivo. Physiol. Genomics, 12, 221-228. Coleman JE, Wu K, Fulle HJ, Semple-Rowland SL (2004) The 5’ flanking sequence of the human retinal guanylyl cyclase 1 gene acquires a photoreceptor cell-restricted activity pattern over the course of retinal development. Mol Vision Oct 10:720-727 <http://www.molvis.org/molvis/v10/a87/>. Williams ML, Coleman JE, Haire SE, Aleman TS, Cideciyan AV, Sokal I, Palczewski K, Jacobson SG, and Semple-Rowland SL (2006) Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) in retina restores vision in an avian model of childhood blindness. PLoS Medicine 3(6):e201. [http://medicine.plosjournals.org/perlserv?request=get-document&doi=10.1371/journal.pmed.0030201] Semple-Rowland SL, Eccles KS, Humberstone EJ (2007) Targeted expression of two proteins in neural retina using self-inactivating, insulated lentiviral vectors carrying two internal independent promoters. Mol Vision 13, 2001-2011. Foster, T.C., Rani, A., Kumar, A., Cui, L. and Semple-Rowland, S.L. (2008) Viral vector mediated delivery of estrogen receptor-alpha to the hippocampus improves spatial learning in adult estrogen receptor-alpha knockout mice. Mol Ther 16, 1587-1593. Semple-Rowland SL, Coggin WE, Geesey M, Eccles K, Abraham L, Pachigar K, Ludlow R, Khani SC, Smith WC (2010) Expression characteristics of dual-promoter lentiviral vectors targeting retinal photoreceptors and Müller cells. Mol Vision 16, 916-934. Verrier, J.D., Semple-Rowland, S., Madorsky, I., Papin, J.E., Notterpek, L. (2010) Reduction of Dicer impairs Schwann cell differentiation and myelination. J Neuroscience Res. 88, 2558-2568. Jackson TC, Verrier JD, Semple-Rowland S, Kumar A, Foster TC. (2010) PHLPP1 splice variants differentially regulate AKT and PKCalpha signaling in hippocampal neurons: Characterization of PHLPP proteins in the adult hippocampus. J Neurochem. 115, 941-955. D. Research Support Ongoing Research Support 2R01 EY011388-11A2 (Susan Semple-Rowland – PI) 12/1/08 - 11/30/2012 NEI/NIH “Rescue of GUCY1*B Phenotype Using Somatic Gene Therapy” The goals of this project are: (1) Develop dual-promoter, self-inactivating, insulated lentiviral vectors capable of delivering multiple therapeutic transgenes to specific retinal cell types and (2) To test the hypothesis that degeneration of photoreceptors that have been treated with targeted corrective gene therapy can be prevented by combining corrective gene therapy with either targeted anti-apoptotic or targeted neurotrophic therapies. Role: PI 9R56 AG037984-11 (Thomas C. Foster – PI) 9/15/2010 - 8/31/2011 “Estrogen and Cognition Over the Lifespan” Examines the hypothesis that estrogen exerts its effects on memory function and delays brain aging by regulating gene transcription through activation of the alpha-estrogen receptor. Role: Co-investigator Completed Research Support 2R01 MH059891 (Thomas C. Foster – PI) NIMH/NIH 3/05/05 - 8/31/2010 “Estrogen and Cognition Over the Lifespan” Examines the hypothesis that estrogen exerts its effects on memory function and delays brain aging by regulating gene transcription through activation of the alpha-estrogen receptor. Role: Co-investigator 5R01 AG014979-11 (Thomas C. Foster -PI) 6/01/07 - 5/31/2012 NIA/NIH “Mechanisms of altered synaptic function during aging” The goals of this project are to understand the basic mechanisms by which synaptic function diminishes with aging and how such changes impact the functions of memory circuits. Role: Co-investigator