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AAV-mediated gene therapy in mouse models of recessive retinal degeneration
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PubMed
title
AAV-mediated gene therapy in mouse models of recessive retinal degeneration
(English)
1 reference
stated in
PubMed
main subject
gene therapy
0 references
retinal degeneration
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based on heuristic
inferred from title
author name string
Pang JJ
series ordinal
1
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stated in
PubMed
Lei L
series ordinal
2
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stated in
PubMed
Dai X
series ordinal
3
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stated in
PubMed
Shi W
series ordinal
4
1 reference
stated in
PubMed
Liu X
series ordinal
5
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stated in
PubMed
Dinculescu A
series ordinal
6
1 reference
stated in
PubMed
McDowell JH
series ordinal
7
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stated in
PubMed
language of work or name
English
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stated in
PubMed
publication date
March 2012
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stated in
PubMed
published in
Current Molecular Medicine
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stated in
PubMed
volume
12
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stated in
PubMed
issue
3
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PubMed
page(s)
316-30
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stated in
PubMed
cites work
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
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PubMed Central
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21 March 2017
Vision 1 year after gene therapy for Leber's congenital amaurosis
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PubMed Central
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21 March 2017
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year
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PubMed Central
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21 March 2017
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
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PubMed Central
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21 March 2017
Safety and efficacy of gene transfer for Leber's congenital amaurosis
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21 March 2017
Usher syndrome: animal models, retinal function of Usher proteins, and prospects for gene therapy
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X-linked retinoschisis: an update
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21 March 2017
Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosis
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PubMed Central
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21 March 2017
In-frame deletion in a novel centrosomal/ciliary protein CEP290/NPHP6 perturbs its interaction with RPGR and results in early-onset retinal degeneration in the rd16 mouse
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Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration
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21 March 2017
Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.
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Synaptogenesis and outer segment formation are perturbed in the neural retina of Crx mutant mice
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21 March 2017
Retinal dehydrogenase 12 (RDH12) mutations in leber congenital amaurosis
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PubMed Central
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21 March 2017
Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue
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PubMed Central
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Channelrhodopsin-2, a directly light-gated cation-selective membrane channel
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PubMed Central
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Mapping of a novel locus for achromatopsia (ACHM4) to 1p and identification of a germline mutation in the alpha subunit of cone transducin (GNAT2)
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PubMed Central
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Mutations in the cone photoreceptor G-protein alpha-subunit gene GNAT2 in patients with achromatopsia
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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Two rhodopsins mediate phototaxis to low- and high-intensity light in Chlamydomonas reinhardtii
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CNGA3 mutations in hereditary cone photoreceptor disorders
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PubMed Central
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21 March 2017
Null RPGRIP1 alleles in patients with Leber congenital amaurosis
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PubMed Central
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Genetic basis of total colourblindness among the Pingelapese islanders
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Recessive mutations in the gene encoding the tubby-like protein TULP1 in patients with retinitis pigmentosa
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A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy
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PubMed Central
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The photoreceptor rim protein is an ABC transporter encoded by the gene for recessive Stargardt's disease (ABCR)
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PubMed Central
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21 March 2017
Total colourblindness is caused by mutations in the gene encoding the alpha-subunit of the cone photoreceptor cGMP-gated cation channel
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PubMed Central
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21 March 2017
Retinitis pigmentosa caused by a homozygous mutation in the Stargardt disease gene ABCR
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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21 March 2017
Retinal degeneration mutants in the mouse
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PubMed Central
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21 March 2017
Update on the molecular genetics of retinitis pigmentosa
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21 March 2017
Mutations in MERTK, the human orthologue of the RCS rat retinal dystrophy gene, cause retinitis pigmentosa
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PubMed Central
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21 March 2017
Insights into the function of Rim protein in photoreceptors and etiology of Stargardt's disease from the phenotype in abcr knockout mice
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Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness
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PubMed Central
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21 March 2017
Effect of gene therapy on visual function in Leber's congenital amaurosis
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Leber congenital amaurosis - a model for efficient genetic testing of heterogeneous disorders: LXIV Edward Jackson Memorial Lecture
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21 March 2017
Leber congenital amaurosis caused by a homozygous mutation (R90W) in the homeodomain of the retinal transcription factor CRX: direct evidence for the involvement of CRX in the development of photoreceptor function
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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21 March 2017
Spectrum of ABCR gene mutations in autosomal recessive macular dystrophies
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PubMed Central
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21 March 2017
De novo mutations in the CRX homeobox gene associated with Leber congenital amaurosis
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PubMed Central
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21 March 2017
Autosomal recessive retinitis pigmentosa and cone-rod dystrophy caused by splice site mutations in the Stargardt's disease gene ABCR
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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21 March 2017
Mutations in RPE65 cause Leber's congenital amaurosis
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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21 March 2017
Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
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21 March 2017
Identification of a photoreceptor-specific mRNA encoded by the gene responsible for retinal degeneration slow (rds)
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21 March 2017
Restoration of cone vision in a mouse model of achromatopsia
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PubMed Central
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21 March 2017
Leber congenital amaurosis linked to AIPL1: a mouse model reveals destabilization of cGMP phosphodiesterase
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21 March 2017
RS-1 Gene Delivery to an Adult Rs1h Knockout Mouse Model Restores ERG b-Wave with Reversal of the Electronegative Waveform of X-Linked Retinoschisis
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21 March 2017
Cone photoreceptor function loss-3, a novel mouse model of achromatopsia due to a mutation in Gnat2
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21 March 2017
The retinitis pigmentosa GTPase regulator (RPGR)- interacting protein: subserving RPGR function and participating in disk morphogenesis
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21 March 2017
Mouse model resources for vision research
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7 April 2017
Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy
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28 September 2017
Gene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice
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28 September 2017
Early-onset, slow progression of cone photoreceptor dysfunction and degeneration in CNG channel subunit CNGB3 deficiency
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28 September 2017
Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis
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28 September 2017
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina
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PubMed Central
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28 September 2017
Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function
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PubMed Central
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28 September 2017
Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.
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PubMed Central
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28 September 2017
Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Replacement gene therapy with a human RPGRIP1 sequence slows photoreceptor degeneration in a murine model of Leber congenital amaurosis
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Achromatopsia as a potential candidate for gene therapy.
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28 September 2017
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.
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28 September 2017
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.
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PubMed Central
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28 September 2017
A homologous genetic basis of the murine cpfl1 mutant and human achromatopsia linked to mutations in the PDE6C gene
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28 September 2017
Impaired cone function and cone degeneration resulting from CNGB3 deficiency: down-regulation of CNGA3 biosynthesis as a potential mechanism
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28 September 2017
Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Homozygosity mapping reveals PDE6C mutations in patients with early-onset cone photoreceptor disorders.
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28 September 2017
Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors
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28 September 2017
Mutations in SPATA7 cause Leber congenital amaurosis and juvenile retinitis pigmentosa
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28 September 2017
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
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28 September 2017
Mutation survey of known LCA genes and loci in the Saudi Arabian population
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28 September 2017
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential
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28 September 2017
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
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28 September 2017
Self-complementary AAV vectors; advances and applications
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28 September 2017
Leber congenital amaurosis: genes, proteins and disease mechanisms
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28 September 2017
AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Rpe65-/- and Lrat-/- mice: comparable models of leber congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Organization and molecular interactions of retinoschisin in photoreceptors
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Mutations in LCA5, encoding the ciliary protein lebercilin, cause Leber congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Two mouse retinal degenerations caused by missense mutations in the beta-subunit of rod cGMP phosphodiesterase gene
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Molecular analysis of the supramolecular usher protein complex in the retina. Harmonin as the key protein of the Usher syndrome
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Prevalence of retinitis pigmentosa in urban and rural adult Chinese: The Beijing Eye Study.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Premature truncation of a novel protein, RD3, exhibiting subnuclear localization is associated with retinal degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Retinal organization in the retinal degeneration 10 (rd10) mutant mouse: a morphological and ERG study
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Usher syndrome: molecular links of pathogenesis, proteins and pathways
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
The nob2 mouse, a null mutation in Cacna1f: anatomical and functional abnormalities in the outer retina and their consequences on ganglion cell visual responses
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Molecular basis of human Usher syndrome: deciphering the meshes of the Usher protein network provides insights into the pathomechanisms of the Usher disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
CNGB3 mutations account for 50% of all cases with autosomal recessive achromatopsia
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Mutations in RDH12 encoding a photoreceptor cell retinol dehydrogenase cause childhood-onset severe retinal dystrophy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Lecithin-retinol acyltransferase is essential for accumulation of all-trans-retinyl esters in the eye and in the liver.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
The nature of dominant mutations of rhodopsin and implications for gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Inherited retinal dystrophy in the rat.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Hereditary X-linked juvenile retinoschisis: a review of the role of Müller cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Complete exon-intron structure of the RPGR-interacting protein (RPGRIP1) gene allows the identification of mutations underlying Leber congenital amaurosis.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Mutations in the gene encoding lecithin retinol acyltransferase are associated with early-onset severe retinal dystrophy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Molecular genetics and prospects for therapy of the inherited retinal dystrophies
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
The mammalian photoreceptor mosaic-adaptive design
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Mutations in the CNGB3 gene encoding the beta-subunit of the cone photoreceptor cGMP-gated channel are responsible for achromatopsia (ACHM3) linked to chromosome 8q21.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
A deletion in a photoreceptor-specific nuclear receptor mRNA causes retinal degeneration in the rd7 mouse
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Selective loss of cone function in mice lacking the cyclic nucleotide-gated channel CNG3.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Morphological, physiological, and biochemical changes in rhodopsin knockout mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Encapsidated adenovirus mini-chromosome-mediated delivery of genes to the retina: application to the rescue of photoreceptor degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
The 220-kDa rim protein of retinal rod outer segments is a member of the ABC transporter superfamily
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Retinal degeneration in the rd mouse is caused by a defect in the beta subunit of rod cGMP-phosphodiesterase
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Retinal receptors in rodents maximally sensitive to ultraviolet light
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 September 2017
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
Channelrhodopsin-2 gene transduced into retinal ganglion cells restores functional vision in genetically blind rats
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
Spectrum of retGC1 mutations in Leber's congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
A novel receptor tyrosine kinase, Mer, inhibits TNF-alpha production and lipopolysaccharide-induced endotoxic shock.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
Different functional outcome of RetGC1 and RPE65 gene mutations in Leber congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
31 May 2018
Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
R91W mutation in Rpe65 leads to milder early-onset retinal dystrophy due to the generation of low levels of 11-cis-retinal
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Restoration of visual response in aged dystrophic RCS rats using AAV-mediated channelopsin-2 gene transfer.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Tool from ancient pharmacopoeia prevents vision loss.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Efficiency of lentiviral transduction during development in normal and rd mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Cone opsin mislocalization in Rpe65-/- mice: a defect that can be corrected by 11-cis retinal
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Retinal degeneration in Aipl1-deficient mice: a new genetic model of Leber congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Cone cell survival and downregulation of GCAP1 protein in the retinas of GC1 knockout mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Evaluation of inner retinal structure in the aged RCS rat.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
In utero gene therapy rescues vision in a murine model of congenital blindness
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Inner retinal abnormalities in a mouse model of Leber's congenital amaurosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Mouse model of subretinal neovascularization with choroidal anastomosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
An RCS-like retinal dystrophy phenotype in mer knockout mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Effect of Rpe65 knockout on accumulation of lipofuscin fluorophores in the retinal pigment epithelium.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
New views on RPE65 deficiency: the rod system is the source of vision in a mouse model of Leber congenital amaurosis.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Prevalence of AIPL1 mutations in inherited retinal degenerative disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Disruption of a retinal guanylyl cyclase gene leads to cone-specific dystrophy and paradoxical rod behavior.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3640500
retrieved
28 November 2018
Retinal degenerations with truncation mutations in the cone-rod homeobox (CRX) gene
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Influence of eye pigmentation and light deprivation on inherited retinal dystrophy in the rat
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Retinal degeneration 6 (rd6): a new mouse model for human retinitis punctata albescens
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
New mouse primary retinal degeneration (rd-3)
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
AAV-mediated gene replacement, either alone or in combination with physical and pharmacological agents, results in partial and transient protection from photoreceptor degeneration associated with betaPDE deficiency
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Prevalence of retinitis pigmentosa in South Indian population aged above 40 years
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Case 15-2009: a man with coma after cardiac arrest
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22300136
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Identifiers
DOI
10.2174/156652412799218877
0 references
PMC publication ID
3640500
1 reference
stated in
PubMed
PubMed publication ID
22300136
1 reference
stated in
PubMed
ResearchGate publication ID
221801028
0 references
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