# (Project 1) Bicistronic Gene Therapies for the Muscular Dystrophies

> **NIH NIH P50** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2024 · $300,029

## Abstract

ABSTRACT
 The goal of this proposal is to complete the remaining pre-clinical proof of concept and safety
studies required to file a pre-IND application with the FDA for a first-in-class bicistronic AAV gene
therapy for patients with Limb Girdle Muscular Dystrophy Type R9 (LGMDR9). Gene therapy has
recently shown great promise in treating some forms of muscular dystrophy as a gene
replacement technology, wherein a normal functional version of the gene mutated in the disease
is introduced back into the muscles of the patient. Such gene replacement technologies are
designed to prevent further development of disease symptoms, but this strategy will do little to
reverse disease in patients where loss of muscle mass and strength is already present. To
accomplish disease reversal for muscular dystrophy patients, a bicistronic, or two gene, therapy
is needed wherein disease prevention is accomplished through gene replacement while a second
gene function is provided to rebuild muscle size and strength. We have developed such a
bicistronic Adeno Associated Virus (AAV) gene therapy for this purpose. Such bicistronic vectors
will be compared to monocistronic (one gene) vectors to demonstrate their superiority in reversing
disease symptoms in a model of LGMDR9 (formerly called LGMD2i). Experiments will be done
to understand the optimal formulation needed for an effective bicistronic gene therapy for
LGMDR9 and also determine the safety of such a treatment. If successful, these experiments
would provide a platform to design new improved therapies almost two dozen other forms of
LGMD and Congenital Muscular Dystrophy (CMD).

## Key facts

- **NIH application ID:** 10992526
- **Project number:** 1P50HD117373-01A1
- **Recipient organization:** RESEARCH INST NATIONWIDE CHILDREN'S HOSP
- **Principal Investigator:** PAUL Taylor MARTIN
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $300,029
- **Award type:** 1
- **Project period:** 2024-08-15 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10992526

## Citation

> US National Institutes of Health, RePORTER application 10992526, (Project 1) Bicistronic Gene Therapies for the Muscular Dystrophies (1P50HD117373-01A1). Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/grant/nih/10992526. Licensed CC0.

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