Article
Long-Term Prevention of Congenital Thrombotic Thrombocytopenic Purpura in ADAMTS13 Knockout Mice by Sleeping Beauty Transposon-Mediated Gene Therapy.
Arteriosclerosis, thrombosis, and vascular biology - 1 May 2017
Verhenne Sebastien, Vandeputte Nele, Pareyn Inge, Izsvák Zsuzsanna, Rottensteiner Hanspeter, Deckmyn Hans, De Meyer Simon F, Vanhoorelbeke Karen
Abstract excerpt
OBJECTIVE: Severe deficiency in the von Willebrand factor-cleaving protease ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin type 1 motif, member 13) because of mutations in the ADAMTS13 gene can lead to acute episodes of congenital thrombotic thrombocytopenic purpura (TTP), requiring prompt treatment. Current treatment consists of therapeutic or prophylactic infusions of fresh frozen plasma....
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