{"title":"Gene Therapy for Hemophilia.","authors":"Peter J Lenting, Sylvia Fong","doi":"10.1182/bloodadvances.2024013864","DOIUrl":null,"url":null,"abstract":"<p><p>Concept Gene therapy with adeno-associated virus (AAV) vectors treats hemophilia A or B by delivering a functional F8 or F9 gene to hepatocytes. This single infusion enables endogenous production of FVIII or FIX protein, reducing bleeding in patients. Evolution Long-term clinical studies have provided insight into the efficacy, safety, and durability of AAV-mediated gene therapies for hemophilia A and B. Gene therapies have now been approved for hemophilia A (valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec; fidanacogene elaparvovec) in select regions. Application The approved gene therapies are associated with a strong reduction in bleeding tendency and increased FVIII or FIX activity levels, without the need for additional FVIII or FIX replacement therapies, in the vast majority of patients. Future Steps Improvements in AAV technology, protein expression, and immunogenicity may render AAV-mediated gene therapies more efficient, longer lasting and safer for people with hemophilia A or B.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":null,"pages":null},"PeriodicalIF":7.4000,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood advances","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/bloodadvances.2024013864","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Concept Gene therapy with adeno-associated virus (AAV) vectors treats hemophilia A or B by delivering a functional F8 or F9 gene to hepatocytes. This single infusion enables endogenous production of FVIII or FIX protein, reducing bleeding in patients. Evolution Long-term clinical studies have provided insight into the efficacy, safety, and durability of AAV-mediated gene therapies for hemophilia A and B. Gene therapies have now been approved for hemophilia A (valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec; fidanacogene elaparvovec) in select regions. Application The approved gene therapies are associated with a strong reduction in bleeding tendency and increased FVIII or FIX activity levels, without the need for additional FVIII or FIX replacement therapies, in the vast majority of patients. Future Steps Improvements in AAV technology, protein expression, and immunogenicity may render AAV-mediated gene therapies more efficient, longer lasting and safer for people with hemophilia A or B.
期刊介绍：
Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016.
Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.
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