Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases

LIN Xue-Qin; WANG Xiao-Le; PENG Jing

doi:10.7499/j.issn.1008-8830.2207055

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Chinese Journal of Contemporary Pediatrics ›› 2022, Vol. 24 ›› Issue (11) : 1281-1287. DOI: 10.7499/j.issn.1008-8830.2207055

REVIEW

Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases

LIN Xue-Qin, WANG Xiao-Le, PENG Jing

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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.

Key words

Lysosomal storage disease / Adeno-associated virus / Gene therapy

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LIN Xue-Qin, WANG Xiao-Le, PENG Jing. Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases[J]. Chinese Journal of Contemporary Pediatrics. 2022, 24(11): 1281-1287 https://doi.org/10.7499/j.issn.1008-8830.2207055

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