FDA approval of Casgevy and Lyfgenia: a dual breakthrough in gene therapies for sickle cell disease.
作者信息
Rahmat Zainab Syyeda, Ali Mohammad Haris, Talha Muhammad, Hasibuzzaman Md Al
机构信息
Faculty of Medicine, Dow Medical College, Dow University of Health Sciences, Karachi.
Shaikh Khalifa Bin Zayed Al-Nahyan Medical and Dental College, Lahore, Pakistan.
出版信息
Ann Med Surg (Lond). 2024 Jul 25;86(9):4966-4968. doi: 10.1097/MS9.0000000000002409. eCollection 2024 Sep.
Abstract
摘要
相似文献
1
FDA approval of Casgevy and Lyfgenia: a dual breakthrough in gene therapies for sickle cell disease.美国食品药品监督管理局批准卡格维（Casgevy）和利夫吉尼亚（Lyfgenia）：镰状细胞病基因疗法的双重突破。
Ann Med Surg (Lond). 2024 Jul 25;86(9):4966-4968. doi: 10.1097/MS9.0000000000002409. eCollection 2024 Sep.
2
Casgevy and Lyfgenia: Two gene therapies for sickle cell disease.卡格维（Casgevy）和利夫吉尼亚（Lyfgenia）：两种治疗镰状细胞病的基因疗法。
Med Lett Drugs Ther. 2024 Jan 22;66(1694):9-10. doi: 10.58347/tml.2024.1694a.
3
Revolutionary breakthrough: FDA approves CASGEVY, the first CRISPR/Cas9 gene therapy for sickle cell disease.革命性突破：美国食品药品监督管理局（FDA）批准了CASGEVY，这是首个用于治疗镰状细胞病的CRISPR/Cas9基因疗法。
Ann Med Surg (Lond). 2024 May 15;86(8):4555-4559. doi: 10.1097/MS9.0000000000002146. eCollection 2024 Aug.
4
Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia.社论：CRISPR-Cas9 治疗性基因编辑治疗镰状细胞病和输血依赖型β-地中海贫血的首次监管批准。
Med Sci Monit. 2024 Mar 1;30:e944204. doi: 10.12659/MSM.944204.
5
Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: A Multidimentional Review.镰状细胞病的造血干细胞移植：多维综述。
Cell Transplant. 2024 Jan-Dec;33:9636897241246351. doi: 10.1177/09636897241246351.
6
Regulatory Assessment of Casgevy for the Treatment of Transfusion-Dependent β-Thalassemia and Sickle Cell Disease with Recurrent Vaso-Occlusive Crises.Casgevy用于治疗依赖输血的β地中海贫血和复发性血管闭塞性危象的镰状细胞病的监管评估。
Curr Issues Mol Biol. 2024 Jul 30;46(8):8209-8225. doi: 10.3390/cimb46080485.
7
Casgevy: Innovative Medicinal Products Require Innovative Approaches to Regulatory Assessment.凯赛维：创新型医药产品需要创新的监管评估方法。
Pharmaceutics. 2024 Jul 6;16(7):906. doi: 10.3390/pharmaceutics16070906.
8
In brief: Casgevy for beta thalassemia.简而言之：用于治疗β地中海贫血的Casgevy。
Med Lett Drugs Ther. 2024 May 13;66(1702):79. doi: 10.58347/tml.2024.1702d.
9
Pharmacists' Knowledge and Perceptions of FDA Approval Standards and the Breakthrough Therapy Designation.药剂师对美国食品药品监督管理局（FDA）批准标准和突破性疗法认定的知识与认知。
Pharmacy (Basel). 2022 Sep 30;10(5):126. doi: 10.3390/pharmacy10050126.
10
Clinical Evidence Supporting FDA Approval of Gene and RNA Therapies for Rare Inherited Conditions.支持 FDA 批准基因和 RNA 疗法治疗罕见遗传性疾病的临床证据。
Paediatr Drugs. 2024 Nov;26(6):741-752. doi: 10.1007/s40272-024-00645-7. Epub 2024 Aug 5.
引用本文的文献
1
Progress in Pseudotyping Lentiviral Vectors Towards Cell-Specific Gene Delivery In Vivo.慢病毒载体假型化用于体内细胞特异性基因递送的研究进展
Viruses. 2025 May 31;17(6):802. doi: 10.3390/v17060802.
本文引用的文献
1
Sickle Cell Disease and Psychosocial Well-Being: Comparison of Patients With Preclinical and Clinical Avascular Necrosis of the Femoral Head.镰状细胞病与心理社会幸福感：股骨头临床前期和临床期无血管性坏死患者的比较。
Turk Arch Pediatr. 2021 Jul 1;56(4):308-315. doi: 10.5152/TurkArchPediatr.2021.20270. eCollection 2021 Jul.
2
l-glutamine, crizanlizumab, voxelotor, and cell-based therapy for adult sickle cell disease: Hype or hope?用于成人镰状细胞病的 l-谷氨酰胺、crizanlizumab、voxelotor 和基于细胞的治疗：炒作还是希望？
Blood Rev. 2022 May;53:100925. doi: 10.1016/j.blre.2021.100925. Epub 2022 Jan 1.
3
βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Anti-sickling Activity in Human Erythroid Cells.βT87Q-珠蛋白基因疗法可减少镰状血红蛋白的产生，从而在人类红系细胞中发挥抗镰变活性。
Mol Ther Methods Clin Dev. 2020 Apr 18;17:912-921. doi: 10.1016/j.omtm.2020.04.013. eCollection 2020 Jun 12.
4
Sickle cell disease: A primary care update.镰状细胞病：初级保健更新。
Cleve Clin J Med. 2020 Jan;87(1):19-27. doi: 10.3949/ccjm.87a.18051. Epub 2020 Jan 2.
5
CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges.CRISPR/Cas9 治疗镰状细胞病：应用、未来可能和挑战。
Adv Exp Med Biol. 2019;1144:37-52. doi: 10.1007/5584_2018_331.
6
Recent advances in globin research using genome-wide association studies and gene editing.利用全基因组关联研究和基因编辑技术在珠蛋白研究方面的最新进展。
Ann N Y Acad Sci. 2016 Mar;1368(1):5-10. doi: 10.1111/nyas.13001. Epub 2016 Feb 11.
7
Optimizing hydroxyurea therapy for sickle cell anemia.优化镰状细胞贫血的羟基脲治疗
Hematology Am Soc Hematol Educ Program. 2015;2015:436-43. doi: 10.1182/asheducation-2015.1.436.
8
Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin.血红蛋白转换的惊人发现：多功能转录因子BCL11A是胎儿血红蛋白的主要抑制因子。
Curr Opin Genet Dev. 2015 Aug;33:62-70. doi: 10.1016/j.gde.2015.08.001. Epub 2015 Sep 14.
9
Global burden of sickle cell anaemia in children under five, 2010-2050: modelling based on demographics, excess mortality, and interventions.2010-2050 年期间五岁以下儿童镰状细胞性贫血的全球负担：基于人口统计学、超额死亡率和干预措施的建模研究。
PLoS Med. 2013;10(7):e1001484. doi: 10.1371/journal.pmed.1001484. Epub 2013 Jul 16.
10
A network model to predict the risk of death in sickle cell disease.一种预测镰状细胞病死亡风险的网络模型。
Blood. 2007 Oct 1;110(7):2727-35. doi: 10.1182/blood-2007-04-084921. Epub 2007 Jun 28.
Zotero 插件