Various AAV Serotypes and Their Applications in Gene Therapy: An Overview

卷:12

Review

关键词: ADENOASSOCIATED VIRUS AAV;    HEPARAN-SULFATE PROTEOGLYCAN;    GLOBOID-CELL LEUKODYSTROPHY;    GROWTH-FACTOR RECEPTOR;    LONG-TERM CORRECTION;    HIGH-EFFICIENCY TRANSDUCTION;    DUCHENNE MUSCULAR-DYSTROPHY;    VASCULAR ENDOTHELIAL-CELLS;    NERVOUS-SYSTEM DISEASE;    ADENO-ASSOCIATED VIRUS;   

DOI  :  10.3390/cells12050785

来源: SCIE

【 摘 要 】

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have no effective treatment. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for various diseases based on adeno-associated viruses (AAVs). Today, many AAV-based gene therapy medications are being investigated in preclinical and clinical trials, and new ones are appearing on the market. In this article, we present a review of AAV discovery, properties, different serotypes, and tropism, and a following detailed explanation of their uses in gene therapy for disease of different organs and systems.