Novel riboswitches regulate AAV delivered transgene expression in mammals via small molecule inducers. ESGCT 2021. X. Guo, et al. Poster Presentation.
AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy for X-Linked Retinitis Pigmentosa Demonstrates Localized Improvements in Static Perimetry ARVO 2022. M. Michaelides et al. Oral Presentation
AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): Human retinal organoid vector efficacy data ARVO 2022. P. Sladen et al. Poster Presentation
RetGC-GUCY2D retinal organoid disease model for AAV gene therapy development. ESGCT 2021. A. Naeem, et al. Poster Presentation.
KCNV2 retinal organoid disease model for KCNV2 AAV gene therapy development. ESGCT 2021. S. Ferrara, et al. Poster Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa Reverses Natural Disease Progression. EURETINA 2021. M. Michaelides, et al. Oral Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 12-month Results From a Phase 1/2 Clinical Trial. AAO 2020. M. Michaelides, et al. Oral Presentation.
AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month Results From a Phase 1/2 Clinical Trial. EURETINA 2020. M. Michaelides, et al. Oral Presentation.
AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 6-month Results From a Phase 1/2 Clinical Trial. ASRS 2020. M. Michaelides, et al. Oral Presentation.
Development and efficacy assessment of AAV2/8-hG1.7p.coCNGA3, a CNGA3 gene therapy vector. ARVO 2019. A. Georgiadis, et al. Poster.
Efficacy and safety of AAV2/5-hRKp.RPGR to treat X-linked retinitis pigmentosa. ESGCT 2017. J. Smith, et al. Poster
Pre-clinical toxicology of AAV2/5-OPTIRPE65, an optimised RPE65 gene therapy vector. ESGCT 2017. A. Georgiadis, et al. Poster
Efficacy assessment and pre-clinical toxicology of AAV2/8-hCARp.hCNGB3, a CNGB3 gene therapy vector. ESGCT 2017. J. 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. A. Georgiadis, et al.
Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa. Gene Therapy, 2015. B. Pawlyk, 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. L. Carvalho, et al.
Gene therapy reduces Parkinson’s disease symptoms by reorganizing functional brain connectivity. Science Translational Medicine, 2018. Martin Niethammer, et al.
Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson’s disease. JCI Insight, 2017. M. Niethammer, et al.
AAV2-GAD gene therapy for advanced Parkinson’s disease: a double-blind, sham-surgery controlled, randomised trial. The Lancet, 2011. P. LeWitt, et al.
Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson’s disease. PNAS, 2007. A. 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. M. Kaplitt, et al.
