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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Association for Research in Vision and Ophthalmology
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7115835/ https://www.ncbi.nlm.nih.gov/pubmed/32724727 http://dx.doi.org/10.1167/tvst.9.7.20 |