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Research Funding Provided by Choroideremia Research Foundation Curechm.Org Research Funding provided by Choroideremia Research Foundation CureCHM.org Funded Researcher Name Institution Project Title USD $ Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2002 Nova Medical School, University of Lisbon, Portugal Choroideremia Research Lab Supplies 1,500 Diseases Research Center Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2003 Nova Medical School, University of Lisbon, Portugal Development of CHM Mouse Model 14,500 Diseases Research Center Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2004 Nova Medical School, University of Lisbon, Portugal Generation of CHM Viral Vector, pt. 1 20,550 Diseases Research Center 2005 Kirill Alexandrov, PhD Max Planck Institute, Germany Forced Expression of REP2 to the Retina 13,000 Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2005 Nova Medical School, University of Lisbon, Portugal Generation of CHM Viral Vector, pt. 2 50,000 Diseases Research Center Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2006 Nova Medical School, University of Lisbon, Portugal Preclinical Gene Therapy Study Year 1 80,460 Diseases Research Center Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2007 Nova Medical School, University of Lisbon, Portugal Preclinical Gene Therapy Study Year 2 69,880 Diseases Research Center Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, Mouse Study Testing for Three Viral Vector 2010 100,000 Ophthalmology University of Pennsylvania, Philadelphia, PA Candidates Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, Alternative In-Vitro Assay to Evaluate Three Viral 2011 75,000 Ophthalmology University of Pennsylvania, Philadelphia, PA Vector Candidates Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2011 Nova Medical School, University of Lisbon, Portugal Pre-Clinical Gene Therapy Study Year 3 90,000 Diseases Research Center Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, Purchase of MP-1 Nidek digital retinal 2012 57,000 Ophthalmology University of Pennsylvania, Philadelphia, PA microperimeter equipment Ataluren to Treat Nonsense-Mediated Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Choroideremia; Evaluate 6 Readthrough 2012 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK Compounds on Zebrafish and iPS Derived CHM Cell 88,864 Ophthalmologist Lines, Fibroblast, RPE with Dr. Kalatzis; Grant 1 Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, First Generation Gene Therapy in Collaboration with 2012 75,000 Ophthalmology University of Pennsylvania Spark Therapeutics, pt. 1 Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic 2013 Diseases Research Center Nova Medical School, University of Lisbon, Portugal Pre-Clinical Gene Therapy Studies pt. 2 90,000 Ataluren to Treat Nonsense-Mediated Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Choroideremia; Evaluate 6 Readthrough 2013 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK 88,864 Compounds on Zebrafish and iPS Derived CHM Cell Ophthalmologist Lines, Fibroblast, RPE with Dr. Kalatzis; Grant 2 Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, First Generation Gene Therapy in Collaboration with 2013 75,000 Ophthalmology University of Pennsylvania, Philadelphia, PA Spark Therapeutics, pt.2 Funded Researcher Name Institution Project Title USD $ Pre-Clinical Gene Therapy Studies for Vasiliki Kalatzis, PhD, Human Genetics, HDR Life Institute for Neurosciences of Montpellier, INSERM, Choroideremia Using a Human Cellular Model: 2013 49,000 Sciences France Differentiation of Patient iPS Cells into Retinal Cells, pt. 2 David Gamm, MD, PhD, Director, McPherson Eye Microscope and Laboratory Equipment for 2013 Research Institute, Associate Professor, Ophthalmology University of Wisconsin, Madison, WI 29,250 Choroideremia Research and Visual Sciences Ian MacDonald, BsC, PhD, Professor of Metabolic An Open Label Clinical Trial of Retinal Gene Therapy 2013 University of Nottingham, UK 22,500 Physiology, Faculty of Medicine & Health Sciences for Choroideremia Ataluren to Treat Nonsense-Mediated Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Choroideremia; Evaluate 6 Readthrough 2014 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK 81,314 Compounds on Zebrafish and iPS Derived CHM Cell Ophthalmologist Lines, Fibroblast, RPE with Dr. Kalatzis; Grant 3 Ataluren to Treat Nonsense-Mediated Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Choroideremia; Evaluate 6 Readthrough 2014 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK 80,846 Compounds on Zebrafish and iPS Derived CHM Cell Ophthalmologist Lines, Fibroblast, RPE with Dr. Kalatzis; Grant 4 Pre-Clinical Gene Therapy Studies for Vasiliki Kalatzis, PhD, Human Genetics, HDR Life Institute for Neurosciences of Montpellier, INSERM, Choroideremia Using a Human Cellular Model: 2014 43,700 Sciences France Differentiation of Patient iPS Cells into Retinal Cells, pt. 1 David Gamm, MD, PhD, Director, McPherson Eye Waisman Center, University of Wisconsin, Madison, The Potential Role of hiPSCs in the Treatment of 2014 Research Institute, Associate Professor, Ophthalmology 55,122 WI Choroideremia and Visual Sciences Jean Bennett, MD, PhD, F.M. Kirby Professor of Scheie Eye Institute, Perelman School of Medicine, 2015 Multi-Focal ERG/Visual Evoked Potentials Machine 34,212 Ophthalmology University of Pennsylvania Mark Pennesi, MD, PhD Assistant Professor in Exploring the Potential of OCT Angiography to 2015 Oregon Health and Science University, Portland, OR 64,350 Ophthalmic Genetics Monitor Progression in Choroideremia Miguel Seabra, MD, PhD, Professor, CEDOC, Chronic Direct Reprogramming of Fibroblasts into Functional 2015 Nova Medical School, University of Lisbon, Portugal 20,000 Diseases Research Center RPE Cells by Specific Transcription Factors 2015 n/a n/a Fight for Sight Partnership - Post-Doctoral Award 23,750 Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Whole Organism Screening for 2015 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK Protective/Regenerative Drug Therapeutics in the 66,471 Ophthalmologist CHM Zebrafish Model Jeffrey S. Mumm, PhD, Helen Larson & Charles Glenn Whole Organism Screening for Wilmer Eye Institute, Johns Hopkins Medicine, 2015 Grover Professor in Ophthalmology, Associate Professor Protective/Regenerative Drug Therapeutics in the 83,527 Baltimore, MD of Ophthalmology CHM Zebrafish Model Gerald Lutty, PhD, Director, Ocular Vasculogenesis and Wilmer Eye Institute, Johns Hopkins Medicine, Production and Testing of CHM hiPSC-Derived 2015 100,000 Angiogenesis Laboratory; Professor of Ophthalmology Baltimore, MD Retinal and Vascular Cells (part 1) Funded Researcher Name Institution Project Title USD $ David Gamm, MD, PhD, Director, McPherson Eye Waisman Center, University of Wisconsin, Madison, 2015 Research Institute; Associate Professor, Ophthalmology Establishment of CHM Biobank 10,050 WI and Visual Sciences David Gamm, MD, PhD, Director, McPherson Eye Waisman Center, University of Wisconsin, Madison, Production and Testing of CHM hiPSC-Derived 2015 Research Institute; Associate Professor, Ophthalmology 100,000 WI Retinal and Vascular Cells (part 2) and Visual Sciences Development of AAV Capsid Variants with Enhanced 2015 n/a 4D Molecular Therapeutics, Emeryville, CA Panrentinal Gene Delivery of the REP-1 Transgene 500,000 for the Treatment of Choroideremia Edwin Stone, MD, PhD, Seamans-Hauser Chair in Molecular Ophthalmology; Director, Molecular Ophthalmology Laboratory; Director, Carver Family Project CHM Genotyping Program (part 2) - Funded 2016 University of Iowa Foundation, Iowa City, IA 55,000 Center for Macular Degeneration; Director, Carver in Conjunction with PTC Therapeutics Nonprofit Genetic Testing Laboratory; Director, Institute for Vision Robert MacLaren, MB, ChB, Dphi, FRCOphth, FRCS, 2016 University of Oxford, UK OPI Lumera OCT Microscope Equipment Purchase 47,000 FACS, FMedSci, Professor of Opthamology Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Whole Organism Screening for 2016 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK Protective/Regenerative Drug Therapeutics in the 28,606 Ophthalmologist CHM Zebrafish Model; grant 2 Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, 2016 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK Freezer for CHM Research Samples 5,788 Ophthalmologist Mariya Moosajee, MBBS, BsC (Hons), PhD, FRCOphth, Investigating the Degenerating Choroid in 2016 Consultant Ophthalmic Surgeon and Clinical Academic University College, London, UK 100,934 Choroideremia Ophthalmologist Michael Young, PhD, FARVO, Co-Director, Ocular Schepens Eye Institute, Mass General Boston; Localized Gene Delivery Through Suprachoroidal 2017 Regenerative Medicine Institute and Director, Minda de Department of Opthamology, Harvard Medical 25,000 Space Using a Novel Autostop Needle Gunzburg Center for Retinal Regeneration School, Boston, MA 50,000 total in David Gamm, MD, PhD, Director, McPherson Eye Year 1: Elucidating the Function of REP1 in Human Waisman Center, University of Wisconsin, Madison, conjunction 2018 Research Institute; Associate Professor, Ophthalmology Pluripotent Stem Cell-Derived RPE and WI with CRF and Visual Sciences Photoreceptor
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