Status and Prospect of Gene Therapy for Hemophilia

VernacularTitle:血友病基因治疗现状与展望
Author: Xinyue DAI ^{1
,

2
,

3} ; Lei ZHANG ^{1
,

2
,

3}
Author Information

1. National Clinical Research Center for Blood Diseases, State Key Laboratory of Experimental Hematology, National Hemophilia Registry Center, Institute of Hematology &
2. Blood Diseases Hospital, Chinese Academy of Medical Sciences &
3. Peking Union Medical College, Tianjin 300020, China.
Publication Type:Journal Article
Keywords: hemophilia; gene therapy; adeno-associated virus
From: JOURNAL OF RARE DISEASES 2022;1(4):386-390
CountryChina
Language:Chinese
Abstract: Hemophilia is a congenital hemorrhagic disease caused by genetic abnormalities in coagulation factor Ⅷ or factor Ⅸ. Current conventional therapy to prevent bleeding requires frequent intravenous injections of coagulation factor concentrates from early childhood. Hemophilia provides an attractive target for gene therapy studies, due to the monogenic nature of these disorders and easily measurable endpoints (factor levels and bleed rates). All successful, pre-clinical and clinical studies to date have utilized recombinant adeno-associated virus(AAV) vectors for factor Ⅷ or Ⅸ hepatocyte transduction. Although this is likely to alter the paradigm of hemophilia care in the near future, it will be important to overcome immune responses against AAV. This review focuses on current successes of clinical trials, and the future direction of hemophilia gene therapy.