腺相关病毒基因治疗血友病。

IF 15.1 1区医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL

Annual review of medicine Pub Date : 2023-01-27 DOI:10.1146/annurev-med-043021-033013

Benjamin J Samelson-Jones, Lindsey A George

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引用次数: 19

摘要

体内基因治疗正迅速成为单基因疾病的一种新的治疗范式。近三十年来，血友病A (HA)和血友病B (HB)一直是基因治疗发展的模式疾病。随着关键腺相关病毒(AAV)载体基因添加试验的完成，这一努力很快就会取得成果，报告了令人鼓舞的结果，目前这一代HA和HB AAV载体有望在不久的将来获得监管部门的批准。在这里，我们回顾了AAV基因治疗HA和HB的临床发展，并检查了最近在血友病和其他单基因疾病的AAV临床试验中出现的突出问题。

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Adeno-Associated Virus Gene Therapy for Hemophilia.

In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy. This effort is soon to bear fruit with completed pivotal adeno-associated viral (AAV) vector gene addition trials reporting encouraging results and regulatory approval widely anticipated in the near future for the current generation of HA and HB AAV vectors. Here we review the clinical development of AAV gene therapy for HA and HB and examine outstanding questions that have recently emerged from AAV clinical trials for hemophilia and other monogenic disorders.

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来源期刊

Annual review of medicine 医学-医学：内科

CiteScore

24.90

自引率

0.00%

发文量

期刊介绍： The Annual Review of Medicine, which has been published since 1950, focuses on important advancements in diverse areas of medicine. These include AIDS/HIV, cardiology, clinical pharmacology, dermatology, endocrinology/metabolism, gastroenterology, genetics, immunology, infectious disease, neurology, oncology/hematology, pediatrics, psychiatry, pulmonology, reproductive medicine, and surgery. The journal's current volume has transitioned from a gated access model to an open access model through the Annual Reviews' Subscribe to Open program. All articles published in the journal are now available under a CC BY license.