Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement - Wikidata - awgldk - TriplyDB

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

http://www.wikidata.org/entity/Q36598421

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Clinical development of gene therapy: results and lessons from recent successes Genetic manipulation for inherited neurodegenerative diseases: myth or reality?Gene therapy on the move Promising and delivering gene therapies for vision loss Gene therapy of inherited retinal degenerations: prospects and challenges Canine retina has a primate fovea-like bouquet of cone photoreceptors which is affected by inherited macular degenerations Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium Biophysical Properties of Optogenetic Tools and Their Application for Vision Restoration Approaches Development of gene and stem cell therapy for ocular neurodegeneration What Is Next for Retinal Gene Therapy?Review: the history and role of naturally occurring mouse models with Pde6b mutations Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.Exciting directions in glaucoma Pharmacological Amelioration of Cone Survival and Vision in a Mouse Model for Leber Congenital Amaurosis Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.Derivation of traceable and transplantable photoreceptors from mouse embryonic stem cells.Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.Rescue of enzymatic function for disease-associated RPE65 proteins containing various missense mutations in non-active sites.Gene Therapy 2017: Progress and Future Directions.Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications.Developments in Ocular Genetics: 2013 Annual Review Translational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations.My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities.A novel form of progressive retinal atrophy in Swedish vallhund dogs Improvement and decline in vision with gene therapy in childhood blindness.Different effects of valproic acid on photoreceptor loss in Rd1 and Rd10 retinal degeneration mice Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.Personalized ophthalmology.The golden era of ocular disease gene discovery: race to the finish.Vector platforms for gene therapy of inherited retinopathies.State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.Pseudo-fovea formation after gene therapy for RPE65-LCA.Clinical characteristics and current therapies for inherited retinal degenerations Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR Extended duration of transgene expression from pegylated POD nanoparticles enables attenuation of photoreceptor degeneration.Genetic deletion of S-opsin prevents rapid cone degeneration in a mouse model of Leber congenital amaurosis Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.Personalized therapeutic strategies for patients with retinitis pigmentosa.Leber congenital amaurosis caused by mutations in RPGRIP1.

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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

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Proceedings of the National Academy of Sciences of the United States of America

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Alejandro J Roman

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Alexander Sumaroka

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András M Komáromy

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Artur V Cideciyan

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Malgorzata Swider

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Melani B Olivares

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Sharon B Schwartz

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Simone Iwabe

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William A Beltran

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P2860

Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis

Gene therapy rescues cone function in congenital achromatopsia.

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue

Effect of gene therapy on visual function in Leber's congenital amaurosis

Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model.

Human cone photoreceptor dependence on RPE65 isomerase

Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.

Lack of fundus autofluorescence to 488 nanometers from childhood on in patients with early-onset severe retinal dystrophy associated with mutations in RPE65.

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Samuel G. Jacobson

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