Restricted accessReplyFirst published online 2019-3
Response to MacLaren et al .,re: Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice. Hum Gene Ther Clin Dev 2018;29(4):188–197
FischerMD, McClementsME, Martinez-Fernandez de la CamaraC, et al.Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa. Mol Ther, 2017; 25:1854–1865.
2.
SongC, ConlonTJ, DengWT, et al.Toxicology and pharmacology of an AAV vector expressing codon-optimized RPGR in RPGR-deficient Rd9 mice. Hum Gene Ther Clin Dev, 2018; 29:188–197.
3.
YeGJ. Codon Optimized Nucleic Acid Encoding a Retinitis Pigmentosa GTPase Regulator (RPGR), WO2015/160893 A1, October 22, 2015.
