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Summary of clinical trials for RPGR-related X-linked retinitis pigmentosa (RP).

Journal: Genes

Article Title: Molecular Strategies for RPGR Gene Therapy

doi: 10.3390/genes10090674

Figure Lengend Snippet: Summary of clinical trials for RPGR-related X-linked retinitis pigmentosa (RP).

Article Snippet: The third trial conducted by Applied Genetic Technologies Corporation (NCT03316560) is using mutated AAV2 capsids (capsids with single tyrosine to phenylalanine (YF) mutations) packaged with full-length, codon optimised human RPGR ORF15 sequence also driven by the rhodopsin kinase promoter (AAV2tYF.GRK1. coRPGR ORF15 ).

Techniques: Clinical Proteomics, Control, Mutagenesis