Correction of ADAMTS13 Deficiency by In Utero Gene Transfer of Lentiviral Vector encoding ADAMTS13 Genes

Deficiency of A Disintegrin And Metalloprotease with ThromboSpondin (ADAMTS13) results in thrombotic thrombocytopenic purpura (TTP). Plasma infusion or exchange is the only effective treatment to date. We show in this study that an administration of a self-inactivating lentiviral vector encoding hum...

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Detalhes bibliográficos
Main Authors: Niiya, Masami, Endo, Masayuki, Shang, Dezhi, Zoltick, Philip W, Muvarak, Nidal E, Cao, Wenjing, Jin, Sheng-Yu, Skipwith, Christopher G, Motto, David G, Flake, Alan W, Zheng, X Long
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2008
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2613173/
https://ncbi.nlm.nih.gov/pubmed/18957966
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.223
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