Mar 4, 2020 · Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in a number of trials across target organs, including eye, liver, skeletal …

Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as hemophilia A and B. ...

Aug 12, 2022 · Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in the last decade and has achieved cures of debilitating diseases such as …

Sep 22, 2022 · Abstract. Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-associated virus (AAV) vector-based gene therapies. Mild to severe …

Jan 20, 2024 · Gene therapy holds promise for patients with inherited monogenic disorders, cancer, and rare genetic diseases. Naturally occurring adeno-associated virus (AAV) offers a …

AAV Vectors and Liver Toxicities: Lessons Learned and Open Questions. Enormous progress has been made from the first liver gene transfer trial for hemophilia B. A deeper understanding of …

As many questions remain on AAV immunogenicity, the field of AAV gene therapy research needs further efforts to resolve the complexity of capsid-related immune responses. The …

Mar 4, 2020 · Potential solutions have been proposed to decrease vector immunogenicity, 152 and it is likely that more effective solutions than corticosteroid administration will be needed to …

Oct 10, 2018 · Since the initial reports of capsid immune responses, 1 our understanding of the impact of AAV immunogenicity has evolved considerably. We now have several tools that can …

Oct 3, 2018 · Since the initial reports of capsid immune responses, 1 our understanding of the impact of AAV immunogenicity has evolved considerably. We now have several tools that can …