AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models - Vasireddy, Vidyullatha; Mills, Jason A.; Gaddameedi, Rajashekhar; Basner-Tschakarjan, Etiena; Kohnke, Monika; Black, Aaron D.; Alexandrov, Krill; Zhou, Shangzhen; Maguire, Albert M.; Chung, Daniel C.; Mac, Helen; Sullivan, Lisa; et al. (PloS one, 2013)
Keywords: Adaptor Proteins, Signal Transducing - genetics; Animal models; Animals; Augmentation; Biology; Blindness; Cell Line; Choroideremia - genetics; Choroideremia - therapy; Degeneration; Dependovirus - genetics; Enzymatic activity; Female; Gene therapy; Genes; Genetic Therapy - adverse effects; Genetic Therapy - methods; Humans; Lymphoblasts; Male; Medicine; Mice; Mutation; Nyctalopia; Patients; Peripheral vision; Physicians; Plasmids - genetics; Pluripotency; Precision Medicine; Protein transport; Protein Transport - genetics; rab GTP-Binding Proteins - metabolism; Recovery of function; Retina; Retinal degeneration; Safety; Stem cells; Toxicity; Viruses; United States--US; Pennsylvania