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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Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: American Society of Hematology 2009
Teme:
Gene Therapy
Online dostop: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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