Novel gene therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A.

IF 0.8 4区医学 Q3 MEDICINE, GENERAL & INTERNAL

Jaapa-Journal of the American Academy of Physician Assistants Pub Date : 2024-11-01 Epub Date: 2024-10-29 DOI:10.1097/01.JAA.0000000000000142

Laura Solano

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.

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镰状细胞病、杜氏肌营养不良症和 A 型血友病的新型基因疗法。

摘要：本文讨论了镰状细胞病、杜氏肌营养不良症和 A 型血友病的新型基因疗法。基因疗法有可能提供更有针对性、更有效的治疗方法，尤其是对于那些获批疗法有限的罕见疾病。本文介绍了首个获得 FDA 批准的 CRISPR/Cas9 疗法，以及四种新型基因疗法的治疗方案、适应症、警告、注意事项、成本和禁忌症。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Jaapa-Journal of the American Academy of Physician Assistants MEDICINE, GENERAL & INTERNAL-

CiteScore

1.20

自引率

9.10%

发文量

310

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