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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Autors principals: 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
Idioma:Inglês
Publicat: American Society of Hematology 2009
Matèries:
Gene Therapy
Accés en línia: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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