{"title":"Novel gene therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A.","authors":"Laura Solano","doi":"10.1097/01.JAA.0000000000000142","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>This article discusses novel genetic therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. Gene therapies have the potential to deliver more targeted and effective approaches to treatment, especially for rare diseases for which the availability of approved therapies is limited. This article describes the first FDA-approved CRISPR/Cas9 treatment and the treatment protocols, indications, warnings, precautions, cost, and contraindications of four novel genetic therapies.</p>","PeriodicalId":48728,"journal":{"name":"Jaapa-Journal of the American Academy of Physician Assistants","volume":null,"pages":null},"PeriodicalIF":0.8000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Jaapa-Journal of the American Academy of Physician Assistants","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1097/01.JAA.0000000000000142","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/10/29 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
引用次数: 0
Abstract
Abstract: This article discusses novel genetic therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. Gene therapies have the potential to deliver more targeted and effective approaches to treatment, especially for rare diseases for which the availability of approved therapies is limited. This article describes the first FDA-approved CRISPR/Cas9 treatment and the treatment protocols, indications, warnings, precautions, cost, and contraindications of four novel genetic therapies.
期刊介绍：
JAAPA is the peer-reviewed clinical journal of the American Academy of Physician Assistants (AAPA). Published for more than 25 years, its mission is to support the ongoing education and advancement of physician assistants (PAs) by publishing current information and research on clinical, health policy, and professional issues.
Published monthly, JAAPA''s award-winning editorial includes:
-Clinical review articles (with AAPA-approved Category I CME in each issue)-
Case reports-
Clinical departments-
Original health services research-
Articles on issues of professional interest to PAs
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
