[Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases]. / è
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Zhongguo Dang Dai Er Ke Za Zhi
; 24(11): 1281-1287, 2022 Nov 15.
Article
en Zh
| MEDLINE
| ID: mdl-36398557
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.
Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Enfermedades por Almacenamiento Lisosomal
/
Dependovirus
Tipo de estudio:
Risk_factors_studies
Límite:
Humans
Idioma:
Zh
Revista:
Zhongguo Dang Dai Er Ke Za Zhi
Año:
2022
Tipo del documento:
Article
País de afiliación:
China