范可尼贫血基因治疗研究进展
Advances in the gene therapy for Fanconi anemia
Received date: 2021-10-20
Online published: 2023-02-16
习必鑫 , 胡群 , 刘爱国 . 范可尼贫血基因治疗研究进展[J]. 临床儿科杂志, 2023 , 41(2) : 156 -160 . DOI: 10.12372/jcp.2023.21e1465
Fanconi anemia is a rare monogenic disease with the hallmark of bone marrow failure. Although allogeneic hematopoietic stem cell transplantation constitutes the preferred therapy for bone marrow failure in Fanconi anemia patients, the increased incidence and mortality of transplant-related complications have seriously affected their quality of life. As medical science advances in recent 30 years, gene therapy may emerge as an innovative low-toxicity therapeutic option for this life-threatening disorder. In this paper, attention is focused on the advances in gene therapy for Fanconi anemia in children.
Key words： Fanconi anemia; gene therapy; clinical research; viral vector
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