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地平线上的希望:镰状细胞病的新型及未来疗法

Hope on the Horizon: New and Future Therapies for Sickle Cell Disease.

作者信息

Kuriri Fahd A

机构信息

Department of Clinical Laboratory Sciences, College of Applied Medical Sciences, Shaqra University, Shaqra, Riyadh 15572, Saudi Arabia.

出版信息

J Clin Med. 2023 Sep 1;12(17):5692. doi: 10.3390/jcm12175692.

Abstract

This article provides an overview of conventional, new, and future treatment options for sickle cell disease (SCD), a genetic disorder affecting the production of hemoglobin. Current treatments include hydroxyurea, a conventional SCD treatment that increases the levels of fetal hemoglobin, and new treatments such as voxelotor, a recently approved SCD treatment that selectively binds hemoglobin, preventing formation of sickled red blood cells. In addition to discussing the mechanisms of action of current SCD treatments, potential side effects are also discussed, highlighting the need for new treatments that can address the limitations of current treatments and improve the quality of life for people with SCD. Future treatments, such as gene therapy, are also explored as promising treatment options for SCD patients.

摘要

本文概述了镰状细胞病(SCD)的传统、新型和未来治疗方案,SCD是一种影响血红蛋白生成的遗传性疾病。目前的治疗方法包括羟基脲,这是一种传统的SCD治疗方法,可提高胎儿血红蛋白水平;还有新型治疗方法,如伏洛托珠单抗,这是一种最近获批的SCD治疗药物,它能选择性地与血红蛋白结合,防止镰状红细胞的形成。除了讨论当前SCD治疗方法的作用机制外,还讨论了潜在的副作用,强调需要新的治疗方法来解决当前治疗方法的局限性,并改善SCD患者的生活质量。还探讨了未来的治疗方法,如基因治疗,作为SCD患者有前景的治疗选择。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/47e3/10488840/520e5c95d725/jcm-12-05692-g001.jpg

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