Dual AAV-based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F
Sehar Riaz, Saumil Sethna, Todd Duncan, Muhammad Azhar Naeem, T. Michael Redmond, Sheikh Riazuddin, Saima Riazuddin, Lívia S. Carvalho, Zubair M. Ahmed
Topics & Concepts
Usher syndromeBiologyRetinitis pigmentosaRetinal pigment epitheliumGenetic enhancementGeneticsProtocadherinGeneCell biologyRetinaNeuroscienceCadherinCellRetinal Development and DisordersPhotoreceptor and optogenetics researchHearing, Cochlea, Tinnitus, Genetics