Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration - Wikidata - wikimedia - TriplyDB

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

http://www.wikidata.org/entity/Q24627838

about

Microphthalmia-associated transcription factor (MITF) promotes differentiation of human retinal pigment epithelium (RPE) by regulating microRNAs-204/211 expression Gene therapy rescues cone function in congenital achromatopsia.Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease The human visual cortex responds to gene therapy-mediated recovery of retinal function Clinical development of gene therapy: results and lessons from recent successes Gene therapy of inherited retinal degenerations: prospects and challenges AAV-mediated gene therapy in mouse models of recessive retinal degeneration Immunology of AAV-Mediated Gene Transfer in the Eye A comprehensive review of retinal gene therapy Restoring the ON Switch in Blind Retinas: Opto-mGluR6, a Next-Generation, Cell-Tailored Optogenetic Tool MicroRNA-restricted transgene expression in the retina CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Bicistronic lentiviruses containing a viral 2A cleavage sequence reliably co-express two proteins and restore vision to an animal model of LCA1 Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer Optogenetic stimulation of the auditory pathway.Gene therapy in the cornea: 2005--present.The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.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.Intravitreal AAV2.COMP-Ang1 Prevents Neurovascular Degeneration in a Murine Model of Diabetic Retinopathy.Assessment of Rod, Cone, and Intrinsically Photosensitive Retinal Ganglion Cell Contributions to the Canine Chromatic Pupillary Response A capillary electrophoresis sequencing method for the identification of mutations in the inverted terminal repeats of adeno-associated virus CRISPR-Cas Genome Surgery in Ophthalmology."To perpetuate blindness!": attitudes of UK patients with inherited retinal disease towards genetic testing.Ab-externo AAV-mediated gene delivery to the suprachoroidal space using a 250 micron flexible microcatheter Gene Therapy 2017: Progress and Future Directions.Interaction of extracellular domain 2 of the human retina-specific ATP-binding cassette transporter (ABCA4) with all-trans-retinal Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications.Rod-derived cone viability factor for treating blinding diseases: from clinic to redox signaling Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.Optogenetic therapy for retinitis pigmentosa.Ocular delivery of macromolecules Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa Long-term gene therapy causes transgene-specific changes in the morphology of regenerating retinal ganglion cells.RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.

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P2860

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

http://www.wikidata.org/entity/Q24627838

description

2009 nî lūn-bûn

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2009 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2009 թվականի դեկտեմբերին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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Gene therapy for Leber's conge ...... rs after vector administration

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Gene therapy for Leber's conge ...... rs after vector administration

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Gene therapy for Leber's conge ...... rs after vector administration

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Molecular Therapy

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Gene therapy for Leber's conge ...... rs after vector administration

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Albert M Maguire

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Carmela Acerra

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Carmela Ziviello

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Federico Mingozzi

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Gui-Shuang Ying

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J Fraser Wright

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Jean Bennett

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Jeannette L Bennicelli

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Jennifer Wellman McDonnell

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Junwei Sun

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P2860

Vision 1 year after gene therapy for Leber's congenital amaurosis

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

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

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer

Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success.

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.

Molecular genetics of Leber congenital amaurosis.

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1288215

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10.1038/MT.2009.277

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3

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18

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Jennifer Wellman McDonnell

Francesca Simonelli

Enrico M. Surace

Francesco Testa

Alberto Auricchio

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2009-12-01T00:00:00Z

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19953081

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