CRISPR/Cas9 nanoparticle enabled therapy for Duchenne Muscular Dystrophy in muscle stem cells

Research Objective

Gene correction of muscle stem cells

Impact

These studies will develop a gene editing based therapy for one of the most prevalent lethal childhood disorders called Duchenne Muscular Dystrophy.

Major Proposed Activities

To identify the best MSNP-CRISPR candidates for CRISPR/Cas9 plasmid delivery in vitro to muscle stem cells
To identify the best MSNP-STEM candidates suitable for delivery of the optimal chemo attractant that enables stem cell migration in vitro
To identify the optimal MSNP-CRISPR and MSNP-STEM candidates from biodistribution studies after systemic injection
To determine the efficiency of MSNP-CRISPR and MSNP-STEM approaches for delivering CRISPR/Cas9 platform to the stem cell niche.
To identify the MSNP delivery strategy that results in restoration of functional dystrophin protein and improved muscle strength after long-term satellite cell correction or reconstitution.

Duchenne Muscular Dystrophy is a progressive muscle wasting disorder with life expectancy of approximately age 20 with incidence of 1 in 5,000 live male births. Because it is a chronic disorder, this disease is devastating to families, involves extensive medical expenses and loss of employment for caregivers. School-age children require a classroom aid and an IEP. A treatment for DMD could reduce health care costs, time lost from work and burden on the public school system.