Gene therapy of inherited retinal degenerations: prospects and challenges (Q27009261)

title

Gene therapy of inherited retinal degenerations: prospects and challenges (English)

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

Sandro Banfi

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1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

Francesca Simonelli

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1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

Enrico M. Surace

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

Alberto Auricchio

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

author name string

Ivana Trapani

1

1 reference

Francesca Simonelli

3

1 reference

Enrico M. Surace

4

1 reference

Alberto Auricchio

5

1 reference

language of work or name

1 reference

April 2015

1 reference

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

volume

26

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5 June 2020

issue

4

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

page(s)

193-200

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

The human visual cortex responds to gene therapy-mediated recovery of retinal function

5 June 2020

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Clinical characteristics and current therapies for inherited retinal degenerations

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

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Gene therapy for inherited retinal degenerations

28 September 2017

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28 September 2017

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28 September 2017

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Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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AAV2 gene therapy readministration in three adults with congenital blindness.

28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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28 September 2017

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Gene delivery to mitotic and postmitotic photoreceptors via compacted DNA nanoparticles results in improved phenotype in a mouse model of retinitis pigmentosa

28 September 2017

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Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

28 September 2017

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Characterization of genome integrity for oversized recombinant AAV vector

28 September 2017

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28 September 2017

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Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous

28 September 2017

1 reference

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Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse

28 September 2017

1 reference

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The role of the photoreceptor ABC transporter ABCA4 in lipid transport and Stargardt macular degeneration

28 September 2017

1 reference

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

28 September 2017

1 reference

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

28 September 2017

1 reference

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Barriers for retinal gene therapy: separating fact from fiction

28 September 2017

1 reference

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Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

28 September 2017

1 reference

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Development of photoreceptor-specific promoters and their utility to investigate EIAV lentiviral vector mediated gene transfer to photoreceptors

28 September 2017

1 reference

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Clinical and molecular genetics of Leber's congenital amaurosis: a multicenter study of Italian patients

28 September 2017

1 reference

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

28 September 2017

1 reference

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

28 September 2017

1 reference

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Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

28 September 2017

1 reference

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Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

28 September 2017

1 reference

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Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy.

28 September 2017

1 reference

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Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

28 September 2017

1 reference

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Molecular genetics of human retinal disease

28 September 2017

1 reference

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

28 September 2017

1 reference

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Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector

28 September 2017

1 reference

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Surfaces of rod photoreceptor disk membranes: light-activated enzymes

28 September 2017

1 reference

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The relationship of primate foveal cones to the pigment epithelium

28 September 2017

1 reference

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Retinal transduction profiles by high-capacity viral vectors

28 September 2017

1 reference

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Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors

31 May 2018

1 reference

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Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Changes in adeno-associated virus-mediated gene delivery in retinal degeneration

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

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

31 May 2018

1 reference

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Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous

31 May 2018

1 reference

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Adeno-associated virus mediated gene therapy for retinal degenerative diseases.

28 November 2018

1 reference

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

28 November 2018

1 reference

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Efficiency of lentiviral transduction during development in normal and rd mice

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Retinal cell type expression specificity of HIV-1-derived gene transfer vectors upon subretinal injection in the adult rat: influence of pseudotyping and promoter.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Lentiviral vector-mediated gene transfer in adult mouse photoreceptors is impaired by the presence of a physical barrier

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Adenoviral-mediated gene transfer to retinal explants during development and degeneration

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Efficient trans-splicing in the retina expands the utility of adeno-associated virus as a vector for gene therapy.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Expression of human factor VIII by splicing between dimerized AAV vectors

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Gene therapy restores vision in a canine model of childhood blindness

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Adenovirus vector-mediated in vivo gene transfer into adult murine retina.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4410187

Case 15-2009: a man with coma after cardiac arrest

28 November 2018

1 reference

12 December 2020

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

1 reference

12 December 2020

The gene therapy journey for hemophilia: are we there yet?

1 reference

12 December 2020

Curing blindness: Vision quest

1 reference

12 December 2020

Identifiers

DOI

10.1089/HUM.2015.030

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

release_4hduuv5irnh6hh667gzyb6mol4

5 June 2020

Fatcat ID

0 references

1 reference

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25762209%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

PubMed publication ID

25762209

2 references