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Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A.

作者信息

Zakas Philip M, Spencer H Trent, Doering Christopher B

机构信息

Graduate Program in Molecular and Systems Pharmacology, Graduate Division of Biological and Biomedical Sciences, Emory University.

Aflac Cancer Center and Blood Disorders Service, Department of Pediatrics, Emory University School of Medicine, Atlanta, Georgia.

出版信息

J Genet Syndr Gene Ther. 2011 Nov 16;1(3). doi: 10.4172/2157-7412.S1-003.

DOI:10.4172/2157-7412.S1-003
PMID:25383239
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4220243/
Abstract
摘要

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Phenotypic correction of hemophilia A in sheep by postnatal intraperitoneal transplantation of FVIII-expressing MSC.通过产后腹腔内移植表达 FVIII 的 MSC 纠正绵羊血友病 A 的表型。
Exp Hematol. 2011 Dec;39(12):1124-1135.e4. doi: 10.1016/j.exphem.2011.09.001. Epub 2011 Sep 8.
2
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.
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Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.基因治疗 X 连锁重症联合免疫缺陷后多克隆 T 细胞 repertoire 的长期持久性。
Sci Transl Med. 2011 Aug 24;3(97):97ra79. doi: 10.1126/scitranslmed.3002715.
4
Site-specific integration and tailoring of cassette design for sustainable gene transfer.位点特异性整合和盒式设计的定制化用于可持续基因转移。
Nat Methods. 2011 Aug 21;8(10):861-9. doi: 10.1038/nmeth.1674.
5
Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugate.经系统给予肽核酸-肽缀合物后对小鼠造血祖细胞进行的靶向基因修饰。
Mol Ther. 2012 Jan;20(1):109-18. doi: 10.1038/mt.2011.163. Epub 2011 Aug 9.
6
In vivo genome editing restores haemostasis in a mouse model of haemophilia.体内基因组编辑恢复血友病小鼠模型的止血功能。
Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177.
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Enhanced biosynthesis of coagulation factor VIII through diminished engagement of the unfolded protein response.通过减少未折叠蛋白反应的结合来增强凝血因子 VIII 的生物合成。
J Biol Chem. 2011 Jul 8;286(27):24451-7. doi: 10.1074/jbc.M111.238758. Epub 2011 May 23.
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Platelet gene therapy improves hemostatic function for integrin alphaIIbbeta3-deficient dogs.血小板基因治疗可改善整合素 αIIbbeta3 缺陷犬的止血功能。
Proc Natl Acad Sci U S A. 2011 Jun 7;108(23):9583-8. doi: 10.1073/pnas.1016394108. Epub 2011 May 23.
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