# Viral vector technology for cell type specific gene delivery

> **NIH NIH R01** · UNIVERSITY OF MINNESOTA · 2022 · $342,667

## Abstract

Project Summary
Gene therapy is a promising treatment for many diseases. For gene therapy to become increasingly successful,
three hurdles must be overcome: We need viral vectors that are (1) safe, (2) efficient, and (3) cell type specific.
Adeno-associated virus (AAV) has emerged as a viral vector that is safe in humans, efficient at delivering
transgenes to both dividing and arrested cells, and able to drive long-term expression. Unfortunately, the broad
tropism of AAV is detrimental when gene delivery to specific cells (e.g., cancer) is paramount and ectopic
expression in healthy cells or tissues poses a risk to the patient’s safety.
We recently reported a working prototype of a novel configurable viral gene delivery technology. This technology
consists of a capsid that we genetically engineer to express an adapter domain to which we covalently attach
monoclonal antibodies to form antibody-AAV composites. AAV tropism is redirected toward the antibody’s
cognate receptor, which is expressed on a targeted cell type, but not off-target cell populations.
Here, we will take the next critical steps to build on this prototype and broaden the impact of our technology. We
will improve composite-AAV formation efficiency and infectivity (Aim 1), comprehensively map additional
engineerable capacity across AAV serotypes identify new capsid engineering strategies and enable machine-
learning guided AAV design (Aim 2) and, as a proof of concept, determine target specificity and spread of AAV
composites in vivo (Aim 3).
The outcome of this work will be a validated viral vector platform technology that uses antibodies to target gene
delivery to rationally identified cell types. This technology will enable fundamentally new gene therapy paradigms
and, in the longer term, lead to new therapeutic approaches for inherited disorders and cancer.

## Key facts

- **NIH application ID:** 10365787
- **Project number:** 1R01GM141152-01A1
- **Recipient organization:** UNIVERSITY OF MINNESOTA
- **Principal Investigator:** Aaron Matthew LeBeau
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $342,667
- **Award type:** 1
- **Project period:** 2022-03-01 → 2026-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10365787

## Citation

> US National Institutes of Health, RePORTER application 10365787, Viral vector technology for cell type specific gene delivery (1R01GM141152-01A1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10365787. Licensed CC0.

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