Adeno-Associated Virus Vectors and Their Application in Gene Therapy Research
Gene therapy,as one of the most cutting-edge fields in biomedicine,has provided a new approach for precision medicine in the treatment of human genetic diseases.Efficient delivery of gene therapy drugs to target tissues or organs through vectors is key to the success of gene therapy.Adeno-associated virus(AAV)has been widely applied in gene therapy due to its high safety,stability,tissue targeting and low immunogenicity.Currently,several gene therapy drugs based on AAV vectors have been marketed,and AAV vectors targeting specific tissues and organs for different diseases are continuously being developed,providing new optional vectors for gene therapy of various diseases in the future.This paper reviews the discovery and development history,biological characteris-tics,vector development and preparation,and key druggability indicators of AAV,summarizes the application of AAV vectors in clinical disease treatment,and looks forward to the possible modification directions of AAV vec-tors,in order to provide references for the development and application of AAV vectors.
NETL
NSTL
万方数据
