Efficacy and safety of AAV2/5-hRKp.RPGR to treat X-linked retinitis pigmentosa. ESGCT 2017. AJ Smith, et al. Poster
Pre-clinical toxicology of AAV2/5-OPTIRPE65, an optimised RPE65 gene therapy vector. ESGCT 2017. Anastasios Georgiadis, et al. Poster Presentation
Efficacy assessment and pre-clinical toxicology of AAV2/8-hCARp.hCNGB3, a CNGB3 gene therapy vector. ESGCT 2017. BJon Telfer, et al. Poster
Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65. Gene Therapy, 2016, Anastasios Georgiadis, et al.
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy. Human Molecular Genetics, 2011, Livia S. Carvalho, et al.
Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson’s disease. JCI Insight, 2017, Martin Niethammer, et al.
AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomised trial. The Lancet, 2011. Prof Peter A. LeWitt, MD, et al.
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson’s disease. PNAS, 2007, Andrew Feigin, et al.
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson’s disease: an open label, phase I trial. The Lancet, 2007. Michael G. Kaplitt, MDPhD, et al.