(Q37956945)

25 August 2017

Gene-based therapies for dominantly inherited retinopathies (English)

1 reference

25 August 2017

G J Farrar

1

1 reference

25 August 2017

S Millington-Ward

2

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25 August 2017

N Chadderton

3

1 reference

25 August 2017

P Humphries

4

1 reference

25 August 2017

P F Kenna

5

1 reference

25 August 2017

17 November 2011

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25 August 2017

1 reference

25 August 2017

19

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25 August 2017

2

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25 August 2017

137-144

1 reference

https://scigraph.springernature.com/pub.10.1038/gt.2011.172

25 August 2017

0 references

cites work

Close genetic linkage between X-linked retinitis pigmentosa and a restriction fragment length polymorphism identified by recombinant DNA probe L1.28

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Identification of disease-causing mutations in autosomal dominant retinitis pigmentosa (adRP) using next-generation DNA sequencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Genetic diagnosis by whole exome capture and massively parallel DNA sequencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Retinitis pigmentosa and allied conditions today: a paradigm of translational research

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

On the genetics of retinitis pigmentosa and on mutation-independent approaches to therapeutic intervention

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Mechanisms of cell death in rhodopsin retinitis pigmentosa: implications for therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Ribozyme uses in retinal gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

RNA interference-mediated suppression and replacement of human rhodopsin in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

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/10.1038%2FGT.2011.172

Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

RNAi-based suppression and replacement of rds-peripherin in retinal organotypic culture

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Specific interaction between the self-splicing RNA of Tetrahymena and its guanosine substrate: implications for biological catalysis by RNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Role of Dicer in posttranscriptional RNA silencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

The silent revolution: RNA interference as basic biology, research tool, and therapeutic

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Interfering with disease: a progress report on siRNA-based therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Zinc-finger protein-targeted gene regulation: genomewide single-gene specificity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Zinc-finger nuclease based genome surgery: it's all about specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Intraocular injection of an aptamer that binds PDGF-B: a potential treatment for proliferative retinopathies

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Adeno-associated virus-mediated anti-DR5 chimeric antibody expression suppresses human tumor growth in nude mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Zinc finger nucleases as tools to understand and treat human diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Tailoring the AAV vector capsid for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Versatility of AAV vectors for retinal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

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/10.1038%2FGT.2011.172

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Transgenic mice carrying the dominant rhodopsin mutation P347S: evidence for defective vectorial transport of rhodopsin to the outer segments

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Stable suppression of tumorigenicity by virus-mediated RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Allele-specific silencing of Alzheimer's disease genes: the amyloid precursor protein genes with Swedish or London mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Targeted disruption of the inosine 5'-monophosphate dehydrogenase type I gene in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Therapeutic benefit derived from RNAi-mediated ablation of IMPDH1 transcripts in a murine model of autosomal dominant retinitis pigmentosa (RP10).

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

The expanding universe of neurotrophic factors: therapeutic potential in aging and age-associated disorders.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Functional cone rescue by RdCVF protein in a dominant model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Depleting Rac1 in mouse rod photoreceptors protects them from photo-oxidative stress without affecting their structure or function

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Induction of molecular chaperones as a therapeutic strategy for the polyglutamine diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

CNTF induces regeneration of cone outer segments in a rat model of retinal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Two animal models of retinal degeneration are rescued by recombinant adeno-associated virus-mediated production of FGF-5 and FGF-18.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Encapsulated cell-based intraocular delivery of ciliary neurotrophic factor in normal rabbit: dose-dependent effects on ERG and retinal histology

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

In contrast to AAV-mediated Cntf expression, AAV-mediated Gdnf expression enhances gene replacement therapy in rodent models of retinal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Increased expression of glutathione peroxidase 4 strongly protects retina from oxidative damage.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

NADPH oxidase plays a central role in cone cell death in retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Identification and characterization of rod-derived cone viability factor

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

The thioredoxin-like protein rod-derived cone viability factor (RdCVFL) interacts with TAU and inhibits its phosphorylation in the retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Neuroprotective effect of overexpression of thioredoxin on photoreceptor degeneration in Tubby mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Misfolded proteins and retinal dystrophies

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Heat shock proteins as suppressors of accumulation of toxic prefibrillar intermediates and misfolded proteins in neurodegenerative diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Heat shock proteins: therapeutic drug targets for chronic neurodegeneration?

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90).

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

Strategems in vitro for gene therapies directed to dominant mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

7 January 2021

Retinopathy induced in mice by targeted disruption of the rhodopsin gene

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

7 January 2021

Generality of the NUX rule: kinetic analysis of the results of systematic mutations in the trinucleotide at the cleavage site of hammerhead ribozymes

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

7 January 2021

Caspase-1 ablation protects photoreceptors in a model of autosomal dominant retinitis pigmentosa

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

7 January 2021

Antioxidants slow photoreceptor cell death in mouse models of retinitis pigmentosa

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.172

7 January 2021

Identifiers

DOI

10.1038/GT.2011.172

1 reference

25 August 2017

1 reference