Built to last: gene therapy for ADA SCID.
作者信息
Pai Sung-Yun
机构信息
Center for Cancer Research, National Cancer Institute, National Institutes of Health.
出版信息
Blood. 2021 Oct 14;138(15):1287-1288. doi: 10.1182/blood.2021012300.
Abstract
摘要
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本文引用的文献
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Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.腺苷脱氨酶严重联合免疫缺陷症的基因治疗后的长期结果。
Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260.
2
Gene Therapies for Primary Immune Deficiencies.原发性免疫缺陷的基因疗法
Front Immunol. 2021 Feb 25;12:648951. doi: 10.3389/fimmu.2021.648951. eCollection 2021.
3
Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.基因修饰干细胞在腺苷脱氨酶缺乏性免疫缺陷中的临床疗效
J Clin Invest. 2017 May 1;127(5):1689-1699. doi: 10.1172/JCI90367. Epub 2017 Mar 27.
4
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.腺苷脱氨酶缺乏症重症联合免疫缺陷的基因治疗：逆转录病毒载体和治疗方案的临床比较。
Blood. 2012 Nov 1;120(18):3635-46. doi: 10.1182/blood-2012-02-400937. Epub 2012 Sep 11.
5
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.腺苷脱氨酶缺乏症严重联合免疫缺陷症的造血干细胞基因治疗可实现长期免疫恢复和代谢纠正。
Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716.
6
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.针对腺苷脱氨酶缺乏所致免疫缺陷的基因治疗。
N Engl J Med. 2009 Jan 29;360(5):447-58. doi: 10.1056/NEJMoa0805817.
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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.通过干细胞基因治疗联合非清髓性预处理纠正腺苷脱氨酶严重联合免疫缺陷症
Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104.
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