Retrospective analysis of death cause in clinical cases of adeno-associated virus gene therapy products
ZHANG Yan-mei
ZHAO Xiao-dong
ZHOU Chang-hui
LI Hua
Abstract：Gene therapy using adeno-associated virus (AAV) vectors has made great progress in the last decade and has enabled the treatment of a number of rare genetic diseases.However,immune responses against AAV capsid antigens or genetically modified products still hinder further progress.This review described the structural characteristics of AAV vector,summarized clinical cases of patient death caused by gene therapy products carried by AAV vectors,described in detail the possible interaction mechanisms and various adverse toxic reaction caused by AAV vectors and gene therapy products,and analyzed the possible death causes of clinical patient combined with these contents.Finally,it is suggested that further research is needed to avoid or reduce the toxicity of immune response to achieve long-term expression of gene therapy.
Keywords：adeno-associated virus vectorsgene therapyinteraction mechanismsimmune responseclinical research
Publication Date：2024-12-31
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：7( 2568-2574 )