Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook. / è
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Zhong Nan Da Xue Xue Bao Yi Xue Ban
; 48(9): 1388-1396, 2023.
Article
in En, Zh
| MEDLINE
| ID: mdl-38044650
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 in treating genetic disorders. The quick approval process for rare diseases medications may bring hope for the treatment of children with rare neurogenetic diseases. AAV-mediated gene therapy is an emerging technology with certain risks and challenges. It is necessary to establish a standardized regulatory system and a sound long-term follow-up system to evaluate the efficacy and safety of gene therapy.
Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Dependovirus
/
Muscular Dystrophy, Duchenne
Limits:
Child
/
Humans
Country/Region as subject:
America do norte
Language:
En
/
Zh
Journal:
Zhong Nan Da Xue Xue Bao Yi Xue Ban
Journal subject:
MEDICINA
Year:
2023
Document type:
Article
Publication country:
CHINA
/
CN
/
REPUBLIC OF CHINA