Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy

• المؤلف الرئيسي: Arun Srivastava (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2023-06-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved. There are now three US Food and Drug Administration (FDA)-approved AAV &qu...