Suppr超能文献

A new frontier: FDA approvals for gene therapy in sickle cell disease.

作者信息

Leonard Alexis, Tisdale John F

机构信息

Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.

Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.

出版信息

Mol Ther. 2024 Feb 7;32(2):264-267. doi: 10.1016/j.ymthe.2024.01.015. Epub 2024 Jan 20.

Abstract
摘要

相似文献

1
A new frontier: FDA approvals for gene therapy in sickle cell disease.
Mol Ther. 2024 Feb 7;32(2):264-267. doi: 10.1016/j.ymthe.2024.01.015. Epub 2024 Jan 20.
2
Approval of the First CRISPR-Cas9 Gene Editing Therapy for Sickle Cell Disease.
Clin Chem. 2024 Oct 3;70(10):1298. doi: 10.1093/clinchem/hvae038.
3
Food and Drug Administration Approval of Glutamine for Sickle Cell Disease: Success and Precautions in Glutamine Research.
JPEN J Parenter Enteral Nutr. 2017 Aug;41(6):912-917. doi: 10.1177/0148607117727271. Epub 2017 Aug 31.
4
Recent and anticipated novel drug approvals for 2023 and 2024.
Am J Health Syst Pharm. 2023 Nov 22;80(23):1729-1732. doi: 10.1093/ajhp/zxad220.
5
Gene therapy targets sickle-cell disease.
Nature. 2018 Dec;564(7735):S12-S13. doi: 10.1038/d41586-018-07646-w.
6
An Ethical and Financial Obligation for Sickle Cell Disease Gene Therapy in the United States.
Ann Intern Med. 2024 Jan;177(1):85-86. doi: 10.7326/M23-2428. Epub 2023 Dec 5.
7
Emerging disease-modifying therapies for sickle cell disease.
Haematologica. 2019 Sep;104(9):1710-1719. doi: 10.3324/haematol.2018.207357. Epub 2019 Aug 14.
8
Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy.
JAMA. 2024 Jan 23;331(4):280. doi: 10.1001/jama.2023.26113.
10
Sickle cell disease: progress towards combination drug therapy.
Br J Haematol. 2021 Jul;194(2):240-251. doi: 10.1111/bjh.17312. Epub 2021 Jan 20.

引用本文的文献

2
Hospital Readmissions Among People With Sickle Cell Disease.
JAMA Netw Open. 2025 Jun 2;8(6):e2517974. doi: 10.1001/jamanetworkopen.2025.17974.
3
Clinical advances in gene, cell, and RNA therapies.
Mol Ther. 2025 Jun 4;33(6):2311. doi: 10.1016/j.ymthe.2025.05.012. Epub 2025 May 20.
4
Comparative Proteomic Analysis Reveals Altered Ciliary Proteins in Sickle Cell Disease.
J Proteome Res. 2025 Jun 6;24(6):2981-2995. doi: 10.1021/acs.jproteome.5c00168. Epub 2025 May 15.
9
Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells.
Mol Ther. 2025 Mar 5;33(3):1014-1030. doi: 10.1016/j.ymthe.2025.01.024. Epub 2025 Jan 14.
10
Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.
Int J Mol Sci. 2024 Jul 4;25(13):7333. doi: 10.3390/ijms25137333.

本文引用的文献

1
Human genetic diversity alters off-target outcomes of therapeutic gene editing.
Nat Genet. 2023 Jan;55(1):34-43. doi: 10.1038/s41588-022-01257-y. Epub 2022 Dec 15.
4
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.
N Engl J Med. 2022 Jan 13;386(2):138-147. doi: 10.1056/NEJMoa2109167. Epub 2021 Dec 12.
5
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease.
Mol Ther Methods Clin Dev. 2021 Oct 1;23:276-285. doi: 10.1016/j.omtm.2021.09.010. eCollection 2021 Dec 10.
7
A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease.
Mol Ther. 2021 Apr 7;29(4):1355-1356. doi: 10.1016/j.ymthe.2021.03.010. Epub 2021 Mar 19.
9
Sickle cell anemia, a molecular disease.
Science. 1949 Apr 29;109(2835):443.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验