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Assessment of AAV Dual Vector Safety in the Abca4(−)(/)(−) Mouse Model of Stargardt Disease

PURPOSE: Adeno-associated viral (AAV) gene therapy treatment for Stargardt disease currently requires a dual vector approach owing to the size of the ATP-binding cassette transporter family member gene (ABCA4). The nature of the dual vector system creates the potential for adverse events. Here we ha...

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Bibliografiska uppgifter
I publikationen:Transl Vis Sci Technol
Huvudupphovsmän: McClements, Michelle E., Barnard, Alun R., Charbel Issa, Peter, MacLaren, Robert E.
Materialtyp: Artigo
Språk:Inglês
Publicerad: The Association for Research in Vision and Ophthalmology 2020
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7115835/
https://ncbi.nlm.nih.gov/pubmed/32724727
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1167/tvst.9.7.20
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