# Novel Therapeutics for Acquired Thrombotic Thrombocytopenic Purpura

> **NIH NIH R01** · UNIVERSITY OF KANSAS MEDICAL CENTER · 2020 · $457,707

## Abstract

Project summary
The overall goal of the proposed study is to understand the mechanism underlying allosteric regulation of
ADAMTS13 function, to determine the therapeutic efficacy of rADAMTS13-loaded platelets in acquired
thrombotic thrombocytopenic purpura (TTP), and to elucidate the mechanism underlying platelet uptake of
rADAMTS13. In Aim 1, we will use the novel deuterium-hydrogen (HX) and mass spectrometry (MS) and other
biophysical tools to determine the role of distal C-terminal domains in allosteric and pH-dependent regulation of
ADAMTS13 function; In Aim 2, we will determine therapeutic efficacy of ADAMTS13-loaded platelets in murine
and human models of acquired TTP; and In Aim 3, we will determine the mechanism underlying platelet
endocytosis of ADAMTS13 by using β3-/-, arf6-/-, and VAMP3-/- mice. We believe that the results of the
proposed study will shed new light on the fundamental biology of ADAMTS13, help to understand the
pathogenesis of acquired TTP, and to develop a novel therapeutic strategy for this fatal syndrome and perhaps
other arterial thrombotic disorders.

## Key facts

- **NIH application ID:** 10200519
- **Project number:** 7R01HL144552-02
- **Recipient organization:** UNIVERSITY OF KANSAS MEDICAL CENTER
- **Principal Investigator:** X. Long Zheng
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $457,707
- **Award type:** 7
- **Project period:** 2019-04-01 → 2023-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10200519

## Citation

> US National Institutes of Health, RePORTER application 10200519, Novel Therapeutics for Acquired Thrombotic Thrombocytopenic Purpura (7R01HL144552-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10200519. Licensed CC0.

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