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Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia
Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for hemophilia. However, immune responses directed against AAV vectors remain a hurdle to the broader use of this gene transfer platform. Both innate and adaptive immune responses can affect the safety and efficacy...
Autores principales: | Monahan, Paul E., Négrier, Claude, Tarantino, Michael, Valentino, Leonard A., Mingozzi, Federico |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8199697/ https://www.ncbi.nlm.nih.gov/pubmed/34199563 http://dx.doi.org/10.3390/jcm10112471 |
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