Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2 - Wikidata - wikimedia - TriplyDB

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

http://www.wikidata.org/entity/Q36906324

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Clinical development of gene therapy: results and lessons from recent successes Manufacturing of recombinant adeno-associated viral vectors for clinical trials Adeno-associated Virus as a Mammalian DNA Vector Promising and delivering gene therapies for vision loss Gene therapy of inherited retinal degenerations: prospects and challenges Immunology of AAV-Mediated Gene Transfer in the Eye AAV2 delivery of Flt23k intraceptors inhibits murine choroidal neovascularization CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Biophysical Properties of Optogenetic Tools and Their Application for Vision Restoration Approaches Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.From caveman companion to medical innovator: genomic insights into the origin and evolution of domestic dogs.Gene Therapy 2017: Progress and Future Directions.Medical management of hereditary optic neuropathies.Improvement and decline in vision with gene therapy in childhood blindness.Vector platforms for gene therapy of inherited retinopathies.Pseudo-fovea formation after gene therapy for RPE65-LCA.Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation Clinical characteristics and current therapies for inherited retinal degenerations Review and update on the molecular basis of Leber congenital amaurosis Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.Development of gene therapy for treatment of age-related macular degeneration.Gene therapy using stem cells Gene therapy for the nervous system: challenges and new strategies.The frequency-response electroretinogram distinguishes cone and abnormal rod function in rd12 mice.Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A AAV-mediated RLBP1 gene therapy improves the rate of dark adaptation in Rlbp1 knockout mice Long-term effect of gene therapy on Leber's congenital amaurosis.Nanoparticle-based technologies for retinal gene therapy Subretinal AAV2.COMP-Ang1 suppresses choroidal neovascularization and vascular endothelial growth factor in a murine model of age-related macular degeneration.Pathogenic mechanisms and the prospect of gene therapy for choroideremia.Prevention of nocturnal elevation of intraocular pressure by gene transfer of dominant-negative RhoA in rats Functional improvement assessed by multifocal electroretinogram after Ocriplasmin treatment for vitreomacular traction.Halting progressive neurodegeneration in advanced retinitis pigmentosa Retinal Gene Therapy: Current Progress and Future Prospects.Prediction of adeno-associated virus neutralizing antibody activity for clinical application.Non-viral therapeutic approaches to ocular diseases: An overview and future directions Multimodal Imaging of Central Retinal Disease Progression in a 2-Year Mean Follow-up of Retinitis Pigmentosa Let There Be Light: Gene and Cell Therapy for Blindness.Hydroxyl PAMAM dendrimer-based gene vectors for transgene delivery to human retinal pigment epithelial cells

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Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

http://www.wikidata.org/entity/Q36906324

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J.OPHTHA.2012.11.048

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Albert M Maguire

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Carmela Acerra

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J Fraser Wright

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Jean Bennett

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Jennifer Wellman

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Junwei Sun

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Kathleen Marshall

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Paolo Melillo

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Settimio Rossi

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P2860

Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial

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

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

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

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

AAV2 gene therapy readministration in three adults with congenital blindness.

Properties of perimetric threshold estimates from Full Threshold, SITA Standard, and SITA Fast strategies.

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

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

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120

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Francesca Simonelli

Enrico M. Surace

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Alberto Auricchio

Katherine A High

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2013-03-06T00:00:00Z

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