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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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| Yayımlandı: | Transl Vis Sci Technol |
|---|---|
| Asıl Yazarlar: | , , , |
| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
The Association for Research in Vision and Ophthalmology
2020
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| Konular: | |
| Online Erişim: | 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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