Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa
about
Biology and therapy of inherited retinal degenerative disease: insights from mouse modelsApplying evolutionary biology to address global challenges.Homozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan.Rod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.CAPN5 gene silencing by short hairpin RNA interference.Use of human pluripotent stem cells to study and treat retinopathiesGeneral pathophysiology in retinal degeneration.Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects.CAPN5 mutation in hereditary uveitis: the R243L mutation increases calpain catalytic activity and triggers intraocular inflammation in a mouse modelHalting progressive neurodegeneration in advanced retinitis pigmentosaBilateral Concordance of the Fundus Hyperautofluorescent Ring in Typical Retinitis Pigmentosa Patients.Therapeutic margins in a novel preclinical model of retinitis pigmentosa.Advances in Gene Therapy for Diseases of the Eye.Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosaAdvances in gene therapy technologies to treat retinitis pigmentosa.Reprogramming metabolism by targeting sirtuin 6 attenuates retinal degeneration.Functional validation of a human CAPN5 exome variant by lentiviral transduction into mouse retina.Gene therapies for inherited retinal disorders.Recent advances of stem cell therapy for retinitis pigmentosa.High-throughput screening assays to identify small molecules preventing photoreceptor degeneration caused by the rhodopsin P23H mutation.Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa.Silencing of tuberin enhances photoreceptor survival and function in a preclinical model of retinitis pigmentosa (an american ophthalmological society thesis).A Novel Retinal Ganglion Cell Promoter for Utility in AAV VectorsGene Therapy Restores Mfrp and Corrects Axial Eye Length.Toward an elucidation of the molecular genetics of inherited retinal degenerations.
P2860
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P2860
Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
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2012年论文
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2012年论文
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name
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@ast
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@en
type
label
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@ast
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@en
prefLabel
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@ast
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@en
P2093
P2860
P356
P1476
Gene therapy provides long-ter ...... model of retinitis pigmentosa
@en
P2093
Javier Sancho-Pelluz
Katherine J Wert
Patsy M Nishina
Richard J Davis
Stephen H Tsang
P2860
P304
P356
10.1093/HMG/DDS466
P577
2012-10-29T00:00:00Z