# Poly(amine-co-ester)s for targeted delivery of gene editing agents to treat cystic fibrosis in animal models: SCGE Disease Models Studies Supplement

> **NIH NIH UH3** · YALE UNIVERSITY · 2022 · $498,197

## Abstract

Project title: Poly(amine-co-ester)s for targeted delivery of gene editing agents to treat cystic fibrosis in animal
models: SCGE Disease Models Studies Supplement
Participating SCGE award, investigators, and institutions: UG3/UH3 HL147352, Poly(amine-co-ester)s for
targeted delivery in vivo of gene editing agents to bone marrow and lung, Yale University, MPI: WM Saltzman
and PM Glazer. Co-I: ME Egan and R Fan
Abstract: Over the past years, with support from the NIH Somatic Cell Gene Editing consortium, we have
developed robust methods for synthesis of a family poly(amine-co-ester) (PACE) polymers that can be
assembled into nanoparticles (NPs) that efficiently entrap nucleic acids. We have shown that these NPs are
effective vectors for delivery of gene editing agents to the lung, and that the efficiency of editing—as well as the
specific lung cell populations that are edited—can be optimized by the selection of optimal combinations of PACE
variants, and by selection of the mode of administration, either intravenous (IV) or intratracheal (IN). Here we
will test these optimized delivery systems for gene editing to treat cystic fibrosis (CF). For that purpose, we have
in house two different, well-characterized animal models of CF: one contains the most common cystic fibrosis
transmembrane conductance regulator (CFTR) gene mutation (F508del) classified as a Class II mutation and
the other contains a common mutation that introduces a stop codon (W1282X) considered a Class I mutation.
Gene editing in these animals will be detected by droplet digital PCR (ddPCR) and deep sequencing, as well as
direct measurement of electrophysiology of the lung and the gut, using approaches we have developed.

## Key facts

- **NIH application ID:** 10619840
- **Project number:** 3UH3HL147352-05S1
- **Recipient organization:** YALE UNIVERSITY
- **Principal Investigator:** PETER M GLAZER
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $498,197
- **Award type:** 3
- **Project period:** 2018-09-07 → 2024-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10619840

## Citation

> US National Institutes of Health, RePORTER application 10619840, Poly(amine-co-ester)s for targeted delivery of gene editing agents to treat cystic fibrosis in animal models: SCGE Disease Models Studies Supplement (3UH3HL147352-05S1). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/10619840. Licensed CC0.

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