2020年3月4日 · 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 muscle, and the central nervous system. Since the initial evidence that AAV vectors can elicit capsid T cell responses in h …

2022年8月12日 · 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. Nevertheless, progress is still being hampered by immune responses against the AAV capsid antigens or the transgene pro …

AAV infections are asymptomatic, and the virus persist for the lifetime of an individual. Nevertheless, AAVs are immunogenic as they stimulate detectable B and T cell responses . As induction of long-lived adaptive immunity depends on antigen-presenting cells that require prior maturation driven by inflammatory signals AAV infections must be ...

2024年1月20日 · This review focuses on aspects of immunity against rAAV, mediated by anti-AAV neutralizing antibodies (NAbs) arising after natural exposure to AAVs or after rAAV vector administration. We provide an in-depth analysis of factors determining AAV seroprevalence and examine clinical approaches to managing anti-AAV NAbs pre- and post-vector ...

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 muscle, and the central nervous system.

2022年9月22日 · Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-associated virus (AAV) vector-based gene therapies. Mild to severe adverse events observed in clinical development have been implicated with host immune responses ...

2023年3月2日 · Multiple factors, including vector design, dose, and route of administration, contribute to the overall immunogenicity of AAVs. The immune responses against the AAV capsid and transgene involve an initial innate sensing.

2018年10月10日 · Gene transfer with adeno-associated virus (AAV) vectors has yielded clinically relevant results in several preclinical and clinical studies, with several candidate treatments proceeding to late-stage clinical trials and two AAV-based gene therapy drugs already approved.

2020年3月4日 · 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 muscle, and the central nervous system.

2018年10月3日 · Gene transfer with adeno-associated virus (AAV) vectors has yielded clinically relevant results in several preclinical and clinical studies, with several candidate treatments proceeding to late-stage clinical trials and two AAV-based gene therapy drugs already approved.