# Novel Vector Platform for Gene Therapy

> **NIH NIH R01** · WASHINGTON UNIVERSITY · 2022 · $370,126

## Abstract

ABSTRACT
Inherited serum deficiency disorders, such as hemophilia and α1-antitrypsin (AAT) deficiency, have been
considered ideal candidates for corrective gene therapy. In this regard, viral vector-mediated transduction of the
liver has been proposed for a number of such disorders wherein hepatocytes represent the normal physiologic
source of the deficient serum factor. Specifically, adeno-associated virus (AAV)-based vectors have
demonstrated utility in hemophilia in animal models and this approach is presently being translated to the context
of human clinical trials. Despite these findings, a number of considerations have led to the recognition that
alternative vector approaches may be required. In the first instance, vector-related liver toxicities make
consideration of non-hepatic sourcing of serum factors desirable. In addition, for some inherited deficiency
disorders, such as AAT deficiency, AAV-mediated in vivo transduction of the liver has not achieved adequate
levels of the deficient serum factor to achieve effective gene therapy. We propose here to test our highly original
approach to accomplish genetic correction of a prototype serum deficiency disorders – AAT deficiency lung
disease. Our technology platform will clearly have broad field impact. In the first regard, we provide the technical
basis for a new translational approaches for the full range of inherited serum deficiency disorders. In the second
regard, we will engineer Ad as a vehicle capable of long term gene expression. This fundamental change in this
important vector's functionalities will dramatically expand the range of utilities whereby it can be employed.

## Key facts

- **NIH application ID:** 10388103
- **Project number:** 5R01EB026468-04
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** David Terry Curiel
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $370,126
- **Award type:** 5
- **Project period:** 2019-07-01 → 2024-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10388103

## Citation

> US National Institutes of Health, RePORTER application 10388103, Novel Vector Platform for Gene Therapy (5R01EB026468-04). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10388103. Licensed CC0.

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