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

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Grant Award Details

Grant Type:
Quest – Discovery Stage Research Projects
Grant Number:
DISC2-08824
Investigator(s):
Name:
Dr. April Pyle Ph.D.
Institution:
University of California, Los Angeles
Type:
PI

Disease Focus:
Muscular Dystrophy, Skeletal/Smooth Muscle disorders
Human Stem Cell Use:
iPS Cell
Award Value:
$2,150,400
Status:
Closed

Progress Reports

Reporting Period:
Year 3 NCE

Grant Application Details

Application Title:

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

Public Abstract:
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.
Statement of Benefit to California:
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.

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