Laura Solano is a genetics professor in the biomedical sciences master's program at A.T. Still University and practices clinically at Pediatric Associates in Fort Worth, Tex. The author has disclosed no potential conflicts of interest, financial or otherwise.
JAAPA. 2024 Nov 1;37(11):17-22. doi: 10.1097/01.JAA.0000000000000142. Epub 2024 Oct 29.
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.
本文讨论了镰状细胞病、杜氏肌营养不良症和血友病 A 的新型基因治疗方法。基因疗法有可能提供更有针对性和更有效的治疗方法，特别是对于那些可用批准疗法有限的罕见疾病。本文描述了首个获得 FDA 批准的 CRISPR/Cas9 治疗方法，以及四种新型基因治疗方法的治疗方案、适应证、警告、注意事项、费用和禁忌证。
