Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho-/- mouse
- Publikationstyp:
- Zeitschriftenaufsatz
- Metadaten:
-
- Autoren
- Arpad Palfi
- Naomi Chadderton
- Mary O'Reilly
- Kerstin Nagel-Wolfrum
- Uwe Wolfrum
- Jean Bennett
- Peter Humphries
- Paul Kenna
- Sophia Millington-Ward
- Jane Farrar
- Autoren-URL
- https://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=fis-test-1&SrcAuth=WosAPI&KeyUT=WOS:000209918700028&DestLinkType=FullRecord&DestApp=WOS_CPL
- DOI
- 10.1038/mtm.2015.16
- eISSN
- 2329-0501
- Externe Identifier
- Clarivate Analytics Document Solution ID: V46YE
- PubMed Identifier: 26029727
- Zeitschrift
- MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
- Artikelnummer
- ARTN 15016
- Datum der Veröffentlichung
- 2015
- Status
- Published
- Titel
- Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho<SUP>-/-</SUP> mouse
- Sub types
- Article
- Ausgabe der Zeitschrift
- 2
Datenquelle: Web of Science (Lite)
- Andere Metadatenquellen:
-
- Autoren
- Arpad Palfi
- Naomi Chadderton
- Mary O'Reilly
- Kerstin Nagel-Wolfrum
- Uwe Wolfrum
- Jean Bennett
- Peter Humphries
- Paul Kenna
- Sophia Millington-Ward
- Jane Farrar
- DOI
- 10.1038/mtm.2015.16
- ISSN
- 2329-0501
- Zeitschrift
- Molecular Therapy - Methods & Clinical Development
- Sprache
- en
- Artikelnummer
- 15016
- Paginierung
- 15016 - 15016
- Datum der Veröffentlichung
- 2015
- Status
- Published
- Herausgeber
- Elsevier BV
- Herausgeber URL
- http://dx.doi.org/10.1038/mtm.2015.16
- Datum der Datenerfassung
- 2018
- Titel
- Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse
- Ausgabe der Zeitschrift
- 2
Datenquelle: Crossref
- Abstract
- As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire of safe and efficient vectors for gene delivery to the target cells. Recombinant adeno-associated virus (AAV) serotype 2/2 has been shown to be well tolerated in the human retina and has provided efficacy in human patients for some inherited retinal degenerations. In this study, the AAV2/8 and AAV2/rh10 serotypes have been compared as a means of gene delivery to mammalian photoreceptor cells using a photoreceptor specific promoter for transgene expression. Both AAV2/8 and AAV2/rh10 provided rescue of the retinal degeneration present in the rhodopsin knockout mouse, with similar levels of benefit as evaluated by molecular, histological, and functional readouts. Transgene expression levels were significantly higher (fivefold) 1 week postsubretinal injection when employing AAV2/8 for rhodopsin gene delivery compared to AAV2/rh10, and were indistinguishable by 6 weeks postadministration of vector. This study reports the use of the AAV2/rh10 serotype to provide rescue in a degenerating retina and provides a comparative evaluation of AAV2/rh10 with respect to AAV2/8, a serotype regarded as providing efficient delivery to photoreceptors.
- Addresses
- Department of Genetics, School of Genetics and Microbiology, Trinity College Dublin , Dublin 2, Ireland.
- Autoren
- Arpad Palfi
- Naomi Chadderton
- Mary O'Reilly
- Kerstin Nagel-Wolfrum
- Uwe Wolfrum
- Jean Bennett
- Peter Humphries
- Paul Kenna
- Sophia Millington-Ward
- Jane Farrar
- DOI
- 10.1038/mtm.2015.16
- eISSN
- 2329-0501
- Externe Identifier
- PubMed Identifier: 26029727
- PubMed Central ID: PMC4444994
- Funding acknowledgements
- NEI NIH HHS: P30 EY001583
- Open access
- true
- ISSN
- 2329-0501
- Zeitschrift
- Molecular therapy. Methods & clinical development
- Sprache
- eng
- Medium
- Electronic-eCollection
- Online publication date
- 2015
- Open access status
- Open Access
- Paginierung
- 15016
- Datum der Veröffentlichung
- 2015
- Status
- Published
- Publisher licence
- CC BY-NC-SA
- Datum der Datenerfassung
- 2015
- Titel
- Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse.
- Sub types
- research-article
- Journal Article
- Ausgabe der Zeitschrift
- 2
Files
http://www.cell.com/article/S2329050116300286/pdf https://europepmc.org/articles/PMC4444994?pdf=render
Datenquelle: Europe PubMed Central
- Abstract
- As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire of safe and efficient vectors for gene delivery to the target cells. Recombinant adeno-associated virus (AAV) serotype 2/2 has been shown to be well tolerated in the human retina and has provided efficacy in human patients for some inherited retinal degenerations. In this study, the AAV2/8 and AAV2/rh10 serotypes have been compared as a means of gene delivery to mammalian photoreceptor cells using a photoreceptor specific promoter for transgene expression. Both AAV2/8 and AAV2/rh10 provided rescue of the retinal degeneration present in the rhodopsin knockout mouse, with similar levels of benefit as evaluated by molecular, histological, and functional readouts. Transgene expression levels were significantly higher (fivefold) 1 week postsubretinal injection when employing AAV2/8 for rhodopsin gene delivery compared to AAV2/rh10, and were indistinguishable by 6 weeks postadministration of vector. This study reports the use of the AAV2/rh10 serotype to provide rescue in a degenerating retina and provides a comparative evaluation of AAV2/rh10 with respect to AAV2/8, a serotype regarded as providing efficient delivery to photoreceptors.
- Date of acceptance
- 2015
- Autoren
- Arpad Palfi
- Naomi Chadderton
- Mary O'Reilly
- Kerstin Nagel-Wolfrum
- Uwe Wolfrum
- Jean Bennett
- Peter Humphries
- Paul Kenna
- Sophia Millington-Ward
- Jane Farrar
- Autoren-URL
- https://www.ncbi.nlm.nih.gov/pubmed/26029727
- DOI
- 10.1038/mtm.2015.16
- Externe Identifier
- PubMed Central ID: PMC4444994
- Funding acknowledgements
- NEI NIH HHS: P30 EY001583
- ISSN
- 2329-0501
- Zeitschrift
- Mol Ther Methods Clin Dev
- Sprache
- eng
- Country
- United States
- Paginierung
- 15016
- PII
- S2329-0501(16)30028-6
- Datum der Veröffentlichung
- 2015
- Status
- Published online
- Datum, an dem der Datensatz öffentlich gemacht wurde
- 2015
- Titel
- Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho(-/-) mouse.
- Sub types
- Journal Article
- Ausgabe der Zeitschrift
- 2
Datenquelle: PubMed
- Beziehungen:
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