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Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved. There are now three US Food and Drug Administration (FDA)-approved AAV “dr...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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American Society of Gene & Cell Therapy
2023
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10244667/ https://www.ncbi.nlm.nih.gov/pubmed/37293185 http://dx.doi.org/10.1016/j.omtn.2023.05.014 |