Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases
LIN Xue-Qin, WANG Xiao-Le, PENG Jing
Department of Pediatrics, Xiangya Hospital, Central South University/Clinical Rearch Center for Children's Neurodevelopmental Disabilities of Hunan Province, Changsha 410008, China
Abstract：Lysosomal storage disorders (LSDs) are a group of single-gene inherited metabolic diseases caused by defects in lysosomal enzymes or function-related proteins. Enzyme replacement therapy is the main treatment method in clinical practice, but it has a poor effect in patients with neurological symptoms. With the rapid development of multi-omics, sequencing technology, and bioengineering, gene therapy has been applied in patients with LSDs. As one of the vectors of gene therapy, adeno-associated virus (AAV) has good prospects in the treatment of genetic and metabolic diseases. More and more studies have shown that AAV-mediated gene therapy is effective in LSDs. This article reviews the application of AAV-mediated gene therapy in LSDs.
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