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Retinal transduction profiles by high-capacity viral vectors
Retinal gene therapy with adeno-associated viral (AAV) vectors is safe and effective in humans. However, the limited cargo capacity of AAV prevents their use for therapy of those inherited retinopathies (IRs) due to mutations in large (>5kb) genes. Viral vectors derived from Adenovirus (Ad), Lent...
Autores principales: | Puppo, Agostina, Cesi, Giulia, Marrocco, Elena, Piccolo, Pasquale, Jacca, Sarah, Shayakhmetov, Dmitry M., Parks, Robin J., Davidson, Beverly L., Colloca, Stefano, Brunetti-Pierri, Nicola, Ng, Philip, Donofrio, Gaetano, Auricchio, Alberto |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4193889/ https://www.ncbi.nlm.nih.gov/pubmed/24989814 http://dx.doi.org/10.1038/gt.2014.57 |
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