Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases.
10.7499/j.issn.1008-8830.2207055
- Author:
Xue-Qin LIN
1
;
Xiao-Le WANG
1
;
Jing PENG
1
Author Information
1. Department of Pediatrics, Xiangya Hospital, Central South University/Clinical Rearch Center for Children's Neurodevelopmental Disabilities of Hunan Province, Changsha 410008, China.
- Publication Type:Journal Article
- Keywords:
Adeno-associated virus;
Gene therapy;
Lysosomal storage disease
- MeSH:
Humans;
Dependovirus/genetics*;
Genetic Therapy/methods*;
Lysosomal Storage Diseases/therapy*;
Enzyme Replacement Therapy;
Proteins/genetics*
- From:
Chinese Journal of Contemporary Pediatrics
2022;24(11):1281-1287
- CountryChina
- Language:Chinese
-
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.
