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Correction of murine ADAMTS13 deficiency by hematopoietic progenitor cell–mediated gene therapy

ADAMTS13, a metalloprotease primarily synthesized in liver and endothelial cells, cleaves von Willebrand factor (VWF) at the central A2 domain, thereby reducing the sizes of circulating VWF multimers. Genetic or acquired deficiency of plasma ADAMTS13 activity leads to a potentially fatal syndrome, t...

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書誌詳細
主要な著者: Laje, Pablo, Shang, Dezhi, Cao, Wenjing, Niiya, Masami, Endo, Masayuki, Radu, Antoneta, DeRogatis, Nicole, Scheiflinger, Friedrich, Zoltick, Philip W., Flake, Alan W., Zheng, X. Long
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Hematology 2009
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC2652365/
https://ncbi.nlm.nih.gov/pubmed/19141866
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2008-08-173021
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