Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa - Wikidata - wikimedia - TriplyDB

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

http://www.wikidata.org/entity/Q36525867

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Biology and therapy of inherited retinal degenerative disease: insights from mouse models Applying 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 retinopathies General 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 model Halting progressive neurodegeneration in advanced retinitis pigmentosa Bilateral 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 pigmentosa Advances 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 Vectors Gene Therapy Restores Mfrp and Corrects Axial Eye Length.Toward an elucidation of the molecular genetics of inherited retinal degenerations.

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Gene therapy provides long-term visual function in a pre-clinical model of retinitis pigmentosa

http://www.wikidata.org/entity/Q36525867

description

2012 nî lūn-bûn

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name

Gene therapy provides long-ter ...... model of retinitis pigmentosa

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Gene therapy provides long-ter ...... model of retinitis pigmentosa

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Human Molecular Genetics

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Gene therapy provides long-ter ...... model of retinitis pigmentosa

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Javier Sancho-Pelluz

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Katherine J Wert

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Patsy M Nishina

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Richard J Davis

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Stephen H Tsang

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P2860

Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy

Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation.

Perspective on genes and mutations causing retinitis pigmentosa.

Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: a morphological and ERG study

Retinal degeneration is rescued in transgenic rd mice by expression of the cGMP phosphodiesterase beta subunit.

New mouse models for recessive retinitis pigmentosa caused by mutations in the Pde6a gene

Functional rescue of degenerating photoreceptors in mice homozygous for a hypomorphic cGMP phosphodiesterase 6 b allele (Pde6bH620Q).

Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer.

Lentivirus-mediated expression of cDNA and shRNA slows degeneration in retinitis pigmentosa.

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558-567

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10.1093/HMG/DDS466

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3

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22

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3542865

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