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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...

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Detalles Bibliográficos
Autores principales: Monahan, Paul E., Négrier, Claude, Tarantino, Michael, Valentino, Leonard A., Mingozzi, Federico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
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