Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system - Wikidata - awgldk - TriplyDB

Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system

Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system

http://www.wikidata.org/entity/Q34898118

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Allotopic expression of mitochondrial-encoded genes in mammals: achieved goal, undemonstrated mechanism or impossible task?Genetic manipulation for inherited neurodegenerative diseases: myth or reality?Leber hereditary optic neuropathy: current perspectives A comprehensive review of retinal gene therapy AAV2 delivery of Flt23k intraceptors inhibits murine choroidal neovascularization Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.Treatment strategies for inherited optic neuropathies: past, present and future Mitochondrial disorders and the eye.Medical management of hereditary optic neuropathies.Adeno-associated virus-mediated gene delivery of the human ND4 complex I subunit in rabbit eyes.The neuro-ophthalmology of mitochondrial disease Nerve fiber layer thinning lags retinal ganglion cell density following crush axonopathy Trial end points and natural history in patients with G11778A Leber hereditary optic neuropathy : preparation for gene therapy clinical trial.Leber's Hereditary Optic Neuropathy-Gene Therapy: From Benchtop to Bedside.Vector platforms for gene therapy of inherited retinopathies.Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.Structural correlation between the nerve fiber layer and retinal ganglion cell loss in mice with targeted disruption of the Brn3b gene.Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1.Animal models of human mitochondrial DNA mutations.Nuclear expression of mitochondrial ND4 leads to the protein assembling in complex I and prevents optic atrophy and visual loss.Nuclear expression of a mitochondrial DNA gene: mitochondrial targeting of allotopically expressed mutant ATP6 in transgenic mice.Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus.The age-regulating protein klotho is vital to sustain retinal function.Correction of Monogenic and Common Retinal Disorders with Gene Therapy.Mitochondrial medicine: to a new era of gene therapy for mitochondrial DNA mutations.Treatment of hereditary optic neuropathies.Novel therapeutic approaches for Leber's hereditary optic neuropathy.Gene therapies for inherited retinal disorders.Emerging aspects of treatment in mitochondrial disorders.Leber Hereditary Optic Neuropathy: Bringing the Lab to the Clinic.Gene and cell-based therapies for inherited retinal disorders: An update.A Review of Mitochondrial Optic Neuropathies: From Inherited to Acquired Forms C57BL/6J, DBA/2J, and DBA/2J.Gpnmb mice have different visual signal processing in the inner retina.Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.The mTORC1 effectors S6K1 and 4E-BP play different roles in CNS axon regeneration.Leber Hereditary Optic Neuropathy: Exemplar of an mtDNA Disease.

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Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system

http://www.wikidata.org/entity/Q34898118

description

2009 nî lūn-bûn

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2009 թուականի Ապրիլին հրատարակուած գիտական յօդուած

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2009 թվականի ապրիլին հրատարակված գիտական հոդված

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2009年の論文

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年学术文章

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2009年學術文章

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Efficiency and safety of AAV-m ...... nit in the mouse visual system

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Efficiency and safety of AAV-m ...... nit in the mouse visual system

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Efficiency and safety of AAV-m ...... nit in the mouse visual system

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Investigative Ophthalmology & Visual Science

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Efficiency and safety of AAV-m ...... nit in the mouse visual system

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John Guy

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Marco Ruggeri

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Rajeshwari D Koilkonda

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Tania Arguello

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Tsung-Han Chou

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Vittorio Porciatti

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Xiaoping Qi

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P2860

Limitations of allotopic expression of mitochondrial genes in mammalian cells

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

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy

Protein import into mitochondria

Parkinson's disease brain mitochondrial complex I has oxidatively damaged subunits and is functionally impaired and misassembled.

In vivo three-dimensional high-resolution imaging of rodent retina with spectral-domain optical coherence tomography.

Assembly of functional proton-translocating ATPase complex in yeast mitochondria with cytoplasmically synthesized subunit 8, a polypeptide normally encoded within the organelle.

Rapid directional shift of mitochondrial DNA heteroplasmy in animal tissues by a mitochondrially targeted restriction endonuclease

Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus.

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10.1167/IOVS.08-3214

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9

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50

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Alfred S. Lewin

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