# Setting the Stage for Replacement of Mitochondrial Genes

> **NIH NIH R01** · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · 2020 · $421,545

## Abstract

Abstract
Setting the Stage for Replacement of Mitochondrial Genes
 The goal of this project is to perform required pre-clinical investigations in a
novel approach to reduce the levels of disease-causing mutant mtDNA in cells from
patients with mitochondrial diseases. We will use mitochondrial-targeted TAL-effector
nucleases (mitoTALENs) as novel gene therapy tools to specifically eliminate selected
mtDNA haplotypes. This approach was successful in cultured cells and now we to use
mitoTALENs in novel ways to alter mtDNA heteroplasmy in mouse tissues, focusing on
expression from rAAV2/9 in limb muscles, extra-ocular muscles and the retina. In
addition, we will optimize a mitoTALEN that we hope can be used in human patients in
the near future.

## Key facts

- **NIH application ID:** 10005433
- **Project number:** 5R01EY010804-25
- **Recipient organization:** UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
- **Principal Investigator:** Carlos Torres Moraes
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $421,545
- **Award type:** 5
- **Project period:** 1994-12-01 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10005433

## Citation

> US National Institutes of Health, RePORTER application 10005433, Setting the Stage for Replacement of Mitochondrial Genes (5R01EY010804-25). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10005433. Licensed CC0.

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