AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura

Severe deficiency of plasma ADAMTS13 activity causes thrombotic thrombocytopenic purpura (TTP), a life-threatening syndrome for which plasma is the only effective therapy currently available. As much as 5% of TTP cases are hereditary, resulting from mutations of the ADAMTS13 gene. Here, we report th...

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Bibliographische Detailangaben
Hauptverfasser: Jin, Sheng-Yu, Xiao, Juan, Bao, Jialing, Zhou, Shangzhen, Wright, J. Fraser, Zheng, X. Long
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Hematology 2013
Schlagworte:
Gene Therapy
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3650704/
https://ncbi.nlm.nih.gov/pubmed/23515928
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2013-02-486779
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