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Allele-specific editing ameliorates dominant retinitis pigmentosa in a transgenic mouse model
Authors:Clarissa Patrizi  Manel Llado  Daniela Benati  Carolina Iodice  Elena Marrocco  Rosellina Guarascio  Enrico M. Surace  Michael E. Cheetham  Alberto Auricchio  Alessandra Recchia
Affiliation:1. Centre for Regenerative Medicine, Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy;2. Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy;3. UCL Institute of Ophthalmology, London EC1V 9EL, UK;4. Medical Genetics, Department of Advanced Biomedicine, Federico II University, 80125 Naples, Italy;5. Medical Genetics, Department of Translational Medicine, Federico II University, 80125 Naples, Italy
Abstract:
Keywords:CRISPR-Cas9 editing  retinitis pigmentosa  Rhodopsin  AAV vector  transgenic mice
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