ArticleBlood2013
AAV-mediated expression of an ADAMTS13 variant prevents shigatoxin-induced thrombotic thrombocytopenic purpura.
Article in Blood, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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Who cites it
29 citing papers in PubMed, 44 citations in OpenAlex.
- Long Term Follow Up of Hereditary Thrombotic Thrombocytopenic Purpura on Plasma Therapy for 9 Years: A Case Report.Clinical case reports · 2026Article
- Rat models of thrombotic thrombocytopenic purpura reveal crucial role of placental ADAMTS13 in perinatal survival.Blood advances · 2026Article
- Preventative and therapeutic effects of a novel humanized anti-glycoprotein Ibα fragment of antigen-binding region in a murine model of thrombotic thrombocytopenic purpura.Journal of thrombosis and haemostasis : JTH · 2025Article
- Novel mechanisms of action of emerging therapies of hereditary thrombotic thrombocytopenic purpura.Expert review of hematology · 2024Review
- Targeting neutrophil extracellular trap accumulation under flow in patients with immune-mediated thrombotic thrombocytopenic purpura.Blood advances · 2024Article
- Hereditary Thrombotic Thrombocytopenic Purpura.Genes · 2023Review
- Animal models for thrombotic thrombocytopenic purpura: a narrative review.Annals of blood · 2023Article
- Clinical Manifestations, Current and Future Therapy, and Long-Term Outcomes in Congenital Thrombotic Thrombocytopenic Purpura.Journal of clinical medicine · 2023Review
- ADAMTS13 conformations and mechanism of inhibition in immune thrombotic thrombocytopenic purpura.Journal of thrombosis and haemostasis : JTH · 2022Review
- TTP: From empiricism for an enigmatic disease to targeted molecular therapies.British journal of haematology · 2022Review
- Plasma Levels of Big Endothelin-1 Are Associated with Renal Insufficiency and In-Hospital Mortality of Immune Thrombotic Thrombocytopenic Purpura.Thrombosis and haemostasis · 2022Article
- Plasma levels of S100A8/A9, histone/DNA complexes, and cell-free DNA predict adverse outcomes of immune thrombotic thrombocytopenic purpura.Journal of thrombosis and haemostasis : JTH · 2021Article
- Histone-induced thrombotic thrombocytopenic purpura inHaematologica · 2020Article
- Zebrafish for thrombocytopoiesis- and hemostasis-related researches and disorders.Blood science (Baltimore, Md.) · 2020Review
- Extracellular DNA-A Danger Signal Triggering Immunothrombosis.Frontiers in immunology · 2020Review
- [Advances in the treatment of thrombotic thrombocytopenic purpura].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2019Article
- Article
- Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Review
- Clinical factors and biomarkers predict outcome in patients with immune-mediated thrombotic thrombocytopenic purpura.Haematologica · 2019Article
- mRNA treatment produces sustained expression of enzymatically active human ADAMTS13 in mice.Scientific reports · 2018Article
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
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 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 × 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. These data support AAV vector-mediated expression of a comparable truncated ADAMTS13 variant as a novel therapeutic approach for hereditary TTP in humans.
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