# Develop combinatorial non-viral and viral CRISPR delivery for lung diseases

> **NIH NIH UG3** · UNIV OF MASSACHUSETTS MED SCH WORCESTER · 2020 · $795,706

## Abstract

Project Summary
The ability to correct disease gene mutations in vivo has broad potential utility for both therapy
and basic research. CRISPR/Cas9 is a powerful RNA-guided tool for genome editing. Our
recent discovery that CRISPR/Cas9 delivery can cure genetic disease in adult mouse liver
provided proof-of-concept of gene correction therapy in vivo. The main goal of this proposal is to
establish innovative delivery technologies to maximize the efficiency of CRISPR delivery and
gene correction in disease-relevant lung cell types. The impact of this project is a novel
paradigm of lung-targeted delivery tools for CRISPR-mediated gene correction. The
development of safe and effective delivery vehicles and genome editing tools will guide future
studies for CRISPR-mediated gene therapy. This project has three aims that focus on different
aspects of lung-directed somatic genome editing: Aim 1: Optimize NP+AAV combination for
delivery and gene correction in mouse lung; Aim 2: Identify the best AAV capsid for lung
delivery and optimize AAV genome as donor template for HDR; Aim 3: Characterize NP+AAV
developed in the UG3 phase in macaque models. This project will develop innovative designs of
adeno-associated virus and nanoparticles to significantly improve the efficiency, cell specificity,
and safety of CRISPR delivery in vivo.

## Key facts

- **NIH application ID:** 9993643
- **Project number:** 5UG3HL147367-03
- **Recipient organization:** UNIV OF MASSACHUSETTS MED SCH WORCESTER
- **Principal Investigator:** DANIEL G ANDERSON
- **Activity code:** UG3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $795,706
- **Award type:** 5
- **Project period:** 2018-09-10 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9993643

## Citation

> US National Institutes of Health, RePORTER application 9993643, Develop combinatorial non-viral and viral CRISPR delivery for lung diseases (5UG3HL147367-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9993643. Licensed CC0.

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