Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook.

ABSTRACT: Rare pediatric neurogenetic diseases always have early onset, no specific therapy, high mortality, and pose a severe risk to the health and survival of children. Adeno-associated virus (AAV)-mediated gene therapy, a type of disease-modifying therapy, provides a new option for the treatment of rare pediatric neurogenetic diseases and represents a significant advancement in the field. Currently, the US Food and Drug Administration (FDA) and the European Medicines Association (EMA) have approved AAV-mediated gene therapy medications for treating spinal muscular atrophy, aromatic L-amino acid decarboxylase deficiency, and Duchenne muscular dystrophy. Numerous preclinical and clinical trial research findings from recent years indicate that AAV-mediated gene therapy has a promising future

SUBMITTER: Wang X 

PROVIDER: S-EPMC10929874 | biostudies-literature | 2023 Sept 28

REPOSITORIES: biostudies-literature