Editing a gene that prompts a cascade of damage after a heart attack appeared to reverse this inevitable course in mice, leaving their hearts remarkably unharmed, a new study by UT Southwestern scientists showed.
Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.
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IMAGE: In a mouse model and in human heart muscle cells, researchers used gene editing to modify specific DNA sequences and restore dystrophin production in mutant dystrophin genes. view more
Credit: UT Southwestern Medical Center
DALLAS - April 30, 2021 - UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients. The approach, described online today in the journal
Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
Study provides new insights into the molecular underpinnings of Duchenne muscular dystrophy
A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in
PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development.
Understanding the activity of genes can shed light on pathologies that affect different tissues in the body. However, says Rhonda Bassel-Duby, Ph.D., a professor of molecular biology at UTSW, studying skeletal muscle has been a challenge because of a key difference from other tissue types; rather than containing a single nucleus that controls the activity of the genes, a skeletal muscle fiber can contain hundreds of nuclei. And it was unknown which genes were activated in all these nuclei, making it unclear
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IMAGE: A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD). view more
Credit: UT Southwestern Medical Center
DALLAS - Dec. 21, 2020 - A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchenne muscular dystrophy (DMD) in humans. The findings, published online recently in
PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development.