[Application of adeno-associated virus-mediated gene therapy in lysosomal storage diseases].
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics(2022)
摘要
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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关键词
Adeno-associated virus,Gene therapy,Lysosomal storage disease
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