Sentences with phrase «from dystrophin»

Clinical trials have used oligonucleotide exon skipping (OEN) to remove mutated exons from the dystrophin transcript.

Using isolated heart cells from dystrophin - deficient mice, the team of Dan Michele, Ph.D., and Joanne Garbincius, of the University of Michigan Department of Molecular & Integrative Physiology, found an explanation for this debilitating protein malfunction — and a potential way to bypass it.

Not exact matches

The inherited condition, most common in boys, results from a lack of dystrophin, a protein that's essential for healthy muscles.

To test the platform, they obtained skin cells from consenting patients at the Center for Duchenne Muscular Dystrophy, all of whom had mutations that fell within the dystrophin gene hot spot.

Once the UCLA researchers had produced iPS cells that were free from Duchenne mutations, they differentiated the iPS cells into cardiac muscle and skeletal muscle cells and then transplanted the skeletal muscle cells into mice that had a genetic mutation in the dystrophin gene.

Patients suffering from Duchenne muscular dystrophy are unable to produce dystrophin.

Dr. Eric Olson (right) shows the dystrophin protein (red) produced in gene - edited heart muscle cells taken from a DMD patient's blood.

The exact mutation varies from patient to patient but in 65 percent of cases, the dystrophin gene is missing large sections of DNA called exons, which carry the instructions for protein production.

This similarity suggests that researchers should target the underlying process in the different varieties of the disease, he says, for example by developing a drug to prevent dystrophin from breaking down.

Muscles from mice treated with modified human stem cells show human dystrophin - producing muscle fibers (yellow) integrated among mouse muscle fibers (red).

It is essential the findings from the study in the dystrophin - deficient mice are confirmed in human DMD patients before its potential applications in these patients can be considered.

Duchenne muscular dystrophy, which strikes in 1 out of 5,000 newborn boys, results from a genetic deficiency in dystrophin, a protein involved in repairing muscle fibers affected by daily movement and activities such as exercise.

The team led by biomedical engineering professor Charles A. Gersbach used a mouse model suffering from a mutated exon of the dystrophin gene, programming CRISPR / CAS9 — a bacterial - protein derived process of cutting and pasting DNA portions — to snip out the defective exon.

Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases.