# Micro-laminin Gene Therapy for MDC1A

> **NIH NIH R21** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2021 · $203,280

## Abstract

SUMMARY/ABSTRACT
Congenital Muscular Dystrophy 1A (MDC1A) is one of the most severe forms of muscular
dystrophy, affecting children at birth and causing dramatic muscle weakness. There are
currently no therapies for MDC1A that can ultimately impact disease outcomes. MDC1A arises
from recessive loss of function mutations in the LAMA2 gene, which encodes laminin a2, the
predominant a chain of laminin in the extracellular matrix (ECM) of skeletal muscle. The
LAMA2 gene is too large to be packaged into Adeno Associated Virus (AAV) vectors, making
gene replacement for MDC1A impossible with the current gold standard used for clinical gene
therapy. We have engineered a micro-laminin gene therapy that can be used with AAV. The
current studies will optimize the therapeutic strength of this micro-laminin gene therapy
approach by engineering in an additional component to build new muscle strength. These
therapies will then be tested in a model for MDC1A. In doing so, this work will develop a single
AAV-mediated gene therapy treatment for patients with MDC1A that has the potential to stop
and reverse the disease process.

## Key facts

- **NIH application ID:** 10198254
- **Project number:** 1R21AR079071-01
- **Recipient organization:** RESEARCH INST NATIONWIDE CHILDREN'S HOSP
- **Principal Investigator:** PAUL Taylor MARTIN
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $203,280
- **Award type:** 1
- **Project period:** 2021-04-15 → 2023-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10198254

## Citation

> US National Institutes of Health, RePORTER application 10198254, Micro-laminin Gene Therapy for MDC1A (1R21AR079071-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10198254. Licensed CC0.

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