Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

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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Bibliographic Details
Main Authors: Paul E. Monahan, Claude Négrier, Michael Tarantino, Leonard A. Valentino, Federico Mingozzi
Format: Article
Language:English
Published: MDPI AG 2021-06-01
Series:Journal of Clinical Medicine
Subjects:
gene therapy
hemophilia
adeno-associated virus vector
genome integration
liver transduction
immunogenicity
Online Access:https://www.mdpi.com/2077-0383/10/11/2471

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