Entering the playing field: Therapy for multiple sulfatase deficiency.
作者信息
Dickson Patricia
机构信息
Department of Pediatrics, Washington University School of Medicine, St. Louis, MO, USA.
出版信息
Mol Ther. 2024 Nov 6;32(11):3756-3757. doi: 10.1016/j.ymthe.2024.10.008. Epub 2024 Oct 30.
Abstract
摘要
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本文引用的文献
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Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.慢病毒基因治疗脑肾上腺脑白质营养不良。
N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442.
2
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.造血干细胞基因疗法改善了多重硫酸酯酶缺乏症临床相关小鼠模型的治疗效果。
Mol Ther. 2024 Nov 6;32(11):3829-3846. doi: 10.1016/j.ymthe.2024.08.015. Epub 2024 Aug 22.
3
Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.逆转录病毒基因治疗腺苷脱氨酶缺乏症的长期和真实世界的安全性和有效性。
Nat Med. 2024 Feb;30(2):488-497. doi: 10.1038/s41591-023-02789-4. Epub 2024 Feb 14.
4
Lysosomal storage disorders: Clinical and therapeutic aspects.溶酶体贮积症：临床与治疗方面。
Handb Clin Neurol. 2023;196:557-567. doi: 10.1016/B978-0-323-98817-9.00006-5.
5
Advances in therapies for neurological lysosomal storage disorders.神经溶酶体贮积症治疗方法的进展
J Inherit Metab Dis. 2023 Sep;46(5):874-905. doi: 10.1002/jimd.12615. Epub 2023 May 2.
6
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.慢病毒造血干细胞基因治疗早发性异染性脑白质营养不良：1/2 期非随机、开放标签、单臂临床试验及扩大使用的长期结果。
Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1.
7
Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.慢病毒造血干细胞基因治疗有益于脑苷脂沉积病。
Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.
8
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation.脑调节对于造血干细胞移植后成功的小胶质细胞重建至关重要。
Proc Natl Acad Sci U S A. 2012 Sep 11;109(37):15018-23. doi: 10.1073/pnas.1205858109. Epub 2012 Aug 23.
9
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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Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease.脐带血移植治疗婴儿型克拉伯病患儿
N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.
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