News Column

Children's Hospital Describes Findings in Thrombocytopenic Purpura

May 29, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Blood Platelet Disorders. According to news reporting from Philadelphia, Pennsylvania, by NewsRx journalists, research stated, "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."

The news correspondents obtained a quote from the research from Children's Hospital, "Here, we report the efficacy and safety of recombinant adeno-associated virus serotype 8 (AAV8)-mediated expression of a murine ADAMTS13 variant (MDTCS), truncated after the spacer domain, in a murine model of TTP. Administration of AAV8-hAAT-mdtcs at doses greater than 2.6 x 10(11) vg/kg body weight resulted in sustained expression of plasma ADAMTS13 activity at therapeutic levels. Expression of the truncated ADAMTS13 variant eliminated circulating ultralarge von Willebrand factor multimers, prevented severe thrombocytopenia, and reduced mortality in Adamts13(-/-) disease-prone mice triggered by shigatoxin-2."

According to the news reporters, the research concluded: "These data support AAV vector-mediated expression of a comparable truncated ADAMTS13 variant as a novel therapeutic approach for hereditary TTP in humans."

For more information on this research see: AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura. Blood, 2013;121(19):3825-9, S1-3. (American Society of Hematology -; Blood -

Our news journalists report that additional information may be obtained by contacting S.Y. Jin, Dept. of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, United States. Additional authors for this research include J. Xiao, J. Bao, S. Zhou, J.F. Wright and X.L Zheng (see also Blood Platelet Disorders).

Keywords for this news article include: Biotechnology, Hematology, Philadelphia, Pennsylvania, Gene Therapy, Therapeutics, United States, Bioengineering, Blood Platelet Disorders, Thrombocytopenic Purpura, North and Central America.

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Source: Gene Therapy Weekly