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Updated: Jun 24, 2025

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血友病基因疗法:另一个祝福还是另一个诅咒?

John Puetz1

  • 1Medical Director of The John Bouhasin Center for Children with Bleeding Disorders and Adjunct Professor, Department of Pediatrics, Division of Pediatric Hematology/Oncology, Saint Louis University School of Medicine and SSM Health Cardinal Glennon Children's Medical Center, St. Louis, Missouri.

Missouri medicine
|June 10, 2024
PubMed
概括

基因疗法提供了对血友病的潜在治疗方法,但临床前研究引发了对基因毒性的担忧. 需要对人类进行长期研究,以确定这种有前途的治疗是否最终是安全和有效的.

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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