Advances in gene therapy for hereditary liver diseases
10.3760/cma.j.issn.1673-4408.2025.09.007
- VernacularTitle:遗传性肝脏疾病基因治疗的研究进展
- Author:
Siyuan FAN
1
;
Hongmei GUO
;
Yu JIN
Author Information
1. 南京医科大学附属儿童医院消化科 210024
- Keywords:
Hereditary liver diseases;
Gene therapy;
Vectors
- From:
International Journal of Pediatrics
2025;52(9):608-613
- CountryChina
- Language:Chinese
-
Abstract:
Hereditary liver diseases refer to liver metabolic disorders caused by gene mutations. The existing clinical treatments are mostly symptomatic therapies，delaying liver damage and improving patients' lives. With the development of gene therapy technology，it has become possible to treat genetic diseases by modifying known gene mutations. This review briefly introduces several common gene therapies（including recombinant adeno-associated virus vectors，CRISPR/Cas9，antisense oligonucleotides，lipid nano particle-mRNA），and discusses the latest research progress，ongoing challenges and potential solutions of gene therapy for hereditary liver diseases through the relevant preclinical studies and drug clinical trials of hereditary liver diseases. In order to promote the further development of gene therapy and provide reference for the treatment of other complex diseases.
