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Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease.评估 AAV 基因治疗在进行性脑白质营养不良小鼠模型中的安全性和早期疗效。
Mol Ther. 2024 Jun 5;32(6):1701-1720. doi: 10.1016/j.ymthe.2024.03.034. Epub 2024 Mar 27.
2
Immunogenicity and toxicity of AAV gene therapy.AAV 基因治疗的免疫原性和毒性。
Front Immunol. 2022 Aug 12;13:975803. doi: 10.3389/fimmu.2022.975803. eCollection 2022.
3
Eukaryotic translation initiation factors as promising targets in cancer therapy.真核翻译起始因子作为癌症治疗的有希望的靶点。
Cell Commun Signal. 2020 Nov 4;18(1):175. doi: 10.1186/s12964-020-00607-9.
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Glial pathology in a novel spontaneous mutant mouse of the Eif2b5 gene: a vanishing white matter disease model.Eif2b5基因新型自发突变小鼠的胶质病理：一种白质消失性疾病模型
J Neurochem. 2020 Jul;154(1):25-40. doi: 10.1111/jnc.14887. Epub 2019 Oct 28.
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High EIF2B5 mRNA expression and its prognostic significance in liver cancer: a study based on the TCGA and GEO database.高EIF2B5 mRNA表达及其在肝癌中的预后意义：基于TCGA和GEO数据库的研究
Cancer Manag Res. 2018 Nov 20;10:6003-6014. doi: 10.2147/CMAR.S185459. eCollection 2018.
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Leukoencephalopathy with vanishing white matter: a review.脑白质消融症：综述。
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Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy.采用肝脏靶向性腺相关病毒2介导的因子IX基因疗法对易产生抑制剂的血友病B犬进行长期校正。
Blood. 2009 Jan 22;113(4):797-806. doi: 10.1182/blood-2008-10-181479. Epub 2008 Oct 28.
8
The spectrum of mutations for the diagnosis of vanishing white matter disease.用于诊断儿童进行性脑白质营养不良的突变谱。
Neurol Sci. 2006 Sep;27(4):271-7. doi: 10.1007/s10072-006-0683-y.
9
EIF2B5 mutations compromise GFAP+ astrocyte generation in vanishing white matter leukodystrophy.EIF2B5突变损害了消失性白质脑白质营养不良中GFAP+星形胶质细胞的生成。
Nat Med. 2005 Mar;11(3):277-83. doi: 10.1038/nm1195. Epub 2005 Feb 20.
10
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus.肌肉注射重组腺相关病毒后人类凝血因子IX的稳定基因转移与表达
Proc Natl Acad Sci U S A. 1997 May 27;94(11):5804-9. doi: 10.1073/pnas.94.11.5804.

Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?

作者信息

Gong Yi, Eichler Florian S

机构信息

Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.

Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.

出版信息

Mol Ther. 2024 Jun 5;32(6):1602-1603. doi: 10.1016/j.ymthe.2024.05.010. Epub 2024 May 21.

DOI:10.1016/j.ymthe.2024.05.010
PMID:38776907
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11184372/
Abstract
摘要