# Sustained delivery of therapeutics to the visual system using genetically modified autologous mesenchymal stem cells to treat blinding diseases

> **NIH NIH R01** · UNIVERSITY OF MISSOURI-COLUMBIA · 2022 · $155,370

## Abstract

PROJECT SUMMARY
The objective of the proposed research is to evaluate the safety and efficacy of a novel method
of delivering therapeutics to the central nervous system (CNS) and the retina to treat diseases
associated with visual impairment. The hypothesis tested is that autologous genetically
modified mesenchymal stem cells (MSCs) can serve as effective vehicles for sustained delivery
of therapeutics to visual centers in the brain and to the retina for the treatment of blinding
diseases that result from pathology in one or more parts of the visual system. Proof of concept
studies will be performed using a well-characterized and validated canine model of CLN2
neuronal ceroid lipofuscinosis, a disease with pediatric onset characterized by widespread
neurodegeneration resulting in progressive loss of vision due to degeneration of both the retina
and visual processing centers in the brain, cognitive and motor decline, and seizures. CLN2
disease is caused by mutations in TPP1, which result in deficiencies of the soluble lysosomal
enzyme tripeptidyl peptidase-1 (TPP1). It is hypothesized that providing TPP1 to the CNS using
implantation of autologous MSCs that have been genetically modified to produce and secrete
the protein will ameliorate disease-related vision loss in the canine model.

## Key facts

- **NIH application ID:** 10558256
- **Project number:** 3R01EY030081-04S1
- **Recipient organization:** UNIVERSITY OF MISSOURI-COLUMBIA
- **Principal Investigator:** Rebecca EH Whiting
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $155,370
- **Award type:** 3
- **Project period:** 2019-05-01 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10558256

## Citation

> US National Institutes of Health, RePORTER application 10558256, Sustained delivery of therapeutics to the visual system using genetically modified autologous mesenchymal stem cells to treat blinding diseases (3R01EY030081-04S1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10558256. Licensed CC0.

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