Titratable and reversible control of CAR-T cell receptor and activity by riboswitch via oral small molecule 2022 ESGCT. Guo et al. Poster Presentation
Novel riboswitches regulate AAV-delivered transgene expression in mammals via oral small molecule inducers 2022 ESGCT. Guo et al. Poster Presentation
Riboswitch-controlled delivery of therapeutic antibodies for gene therapy 2022 ESGCT. Guo et al. Poster Presentation
Riboswitch-controlled delivery of therapeutic hormones for gene therapy Session Title: Metabolic diseases 2022 ESGCT. Guo et al. Poster Presentation
Novel riboswitches regulate AAV delivered transgene expression in mammals via small molecule inducers. ESGCT 2021. Guo et al. Poster Presentation.
Promoter Engineering Platform at MeiraGTx 2022 ESGCT. Liu et al. Poster Presentation
AI-driven promoter optimization at MeiraGTx 2022 ESGCT. Liu et al. Poster Presentation
A CNS-targeted gene therapy for the treatment of obesity 2022 ESGCT. Sullivan, et al. Poster Presentation
Development of an in-vitro testing platform for AAV-UPF1 gene therapy to treat ALS 2022 ESGCT. Gordon et al. Poster Presentation
Development of optimized ATP7B gene therapy vectors for the treatment of Wilson’s Disease with increased potency 2022 ESGCT. Sullivan et al. Poster Presentation
Multivariate analysis for increased understanding of MeiraGTx upstream process 2022 ESGCT. Valinhas et al. Poster Presentation
Improving AAV in vitro transducibility for cell-based potency assay development 2022 ESGCT. Georgiadis, et al. Poster Presentation
Use of anion exchange chromatography to provide high empty AAV capsid removal and product yields 2022 ESGCT. Senaratne et al. Poster Presentation
Designing and screening formulations to improve manufacturability and distribution of AAV gene therapies 2022 ESGCT. Senaratne et al. Poster Presentation
Optimization and scale-up of AAV2-AQP1 production using TransIT-VirusGEN 2022 ESGCT. Dzopa et al. Poster Presentation
Ph1/2 AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy Trial in RPGR-associated X-linked Retinitis Pigmentosa (XLRP) AAO 2022. M. Michaelides et al. Oral 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.
UPF1 delivered by novel expression-enhanced promoters protects cultured neurons in a genetic ALS model 2022 ESGCT. Liu et al. Poster Presentation
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.
