Gene therapy for inherited retinal and optic nerve degenerations. (Q45873452)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

title

Gene therapy for inherited retinal and optic nerve degenerations (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

0 references

Nicholas A Moore

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

Peter Bracha

series ordinal

1 reference

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7 April 2020

language of work or name

English

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publication date

23 October 2017

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

Expert Opinion on Biological Therapy

7 April 2020

published in

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7 April 2020

volume

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7 April 2020

issue

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7 April 2020

page(s)

37-49

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

Prevalence of mutations causing retinitis pigmentosa and other inherited retinopathies

7 April 2020

cites work

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene and cell-based therapies for inherited retinal disorders: An update

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Advances in AAV vector development for gene therapy in the retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy using adeno-associated virus vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Self-complementary AAV vectors; advances and applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Retroviral vector interactions with hematopoietic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Lentiviral vectors: basic to translational

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Systematic determination of the packaging limit of lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Ocular immune privilege: therapeutic opportunities from an experiment of nature

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy for age-related macular degeneration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Genetic manipulation for inherited neurodegenerative diseases: myth or reality?

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Leber congenital amaurosis: comprehensive survey of the genetic heterogeneity, refinement of the clinical definition, and genotype-phenotype correlations as a strategy for molecular diagnosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Leber congenital amaurosis: genes, proteins and disease mechanisms

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Leber congenital amaurosis: a genetic paradigm

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Safety and efficacy of gene transfer for Leber's congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Long-term effect of gene therapy on Leber's congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Improvement and decline in vision with gene therapy in childhood blindness.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

A Comprehensive Analysis of Choroideremia: From Genetic Characterization to Clinical Practice

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Rab escort protein 1 (REP1) in intracellular traffic: a functional and pathophysiological overview.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Pathogenic mechanisms and the prospect of gene therapy for choroideremia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Organization of the Rab-GDI/CHM superfamily: the functional basis for choroideremia disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Recent advances and future prospects in choroideremia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

How sensitive to clinical change are ETDRS logMAR visual acuity measurements?

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Visual Acuity after Retinal Gene Therapy for Choroideremia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Retinal pigment epithelium defects accelerate photoreceptor degeneration in cell type-specific knockout mouse models of choroideremia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Evaluation of retinal photoreceptors and pigment epithelium in a female carrier of choroideremia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Nonsense mutation in MERTK causes autosomal recessive retinitis pigmentosa in a consanguineous Pakistani family

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Mutation of the receptor tyrosine kinase gene Mertk in the retinal dystrophic RCS rat

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene-based therapies for dominantly inherited retinopathies

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Expression in cochlea and retina of myosin VIIa, the gene product defective in Usher syndrome type 1B

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete cystic fibrosis transmembrane regulator reconstitution

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

The ATP-binding cassette transporter ABCA4: structural and functional properties and role in retinal disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy for Stargardt disease associated with ABCA4 gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

The optimized allotopic expression of ND1 or ND4 genes restores respiratory chain complex I activity in fibroblasts harboring mutations in these genes

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Re: Feuer et al.: Gene therapy for Leber hereditary optic neuropathy: initial results (Ophthalmology 2016;123:558-570).

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Efficacy and Safety of rAAV2-ND4 Treatment for Leber's Hereditary Optic Neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Achromatopsia: on the doorstep of a possible therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy for achromatopsia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Clinical utility gene card for: Achromatopsia - update 2013.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Restoration of cone vision in a mouse model of achromatopsia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Gene therapy rescues cone function in congenital achromatopsia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Restoration of synaptic function in sight for degenerative retinal disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

X-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Inactivation of the murine X-linked juvenile retinoschisis gene, Rs1h, suggests a role of retinoschisin in retinal cell layer organization and synaptic structure

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Disease mechanisms and gene therapy in a mouse model for X-linked retinoschisis

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Retinoschisin gene therapy in photoreceptors, Müller glia or all retinal cells in the Rs1h-/- mouse.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

New recombinant serotypes of AAV vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

AAV's anatomy: roadmap for optimizing vectors for translational success

21 January 2018

1 reference

https://api.crossref.org/works/10.1080%2F14712598.2018.1389886

10.1080/14712598.2018.1389886

21 January 2018

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json

7 April 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29057663%20AND%20SRC:MED&resulttype=core&format=json