Sentences with phrase «corrected duchenne»

Using the new gene - editing enzyme CRISPR - Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.

The UT Southwestern group had previously used CRISPR - Cas9, the original gene - editing system, to correct the Duchenne defect in a mouse model of the disease and in human cells.

«By either skipping a mutation region or precisely repairing a mutation in the gene, CRISPR - Cpf1 - mediated genome editing not only corrects Duchenne muscular dystrophy mutations but also improves muscle contractility and strength,» said co-author Dr. Rhonda Bassel - Duby, Professor of Molecular Biology and Associate Director of the Hamon Center for Regenerative Science and Medicine.

«Gene - editing alternative corrects Duchenne muscular dystrophy.»

Not exact matches

And Crispr - Cas9 isn't even the only type of Crispr out there: On April 12, researchers at the University of Texas Southwestern Medical Center announced they had successfully paired the gene - editing tool with a different kind of enzyme, called Cpf1, to correct mutations associated with the devastating muscle - wasting disorder Duchenne muscular dystrophy.

Researchers from Duke University had previously used CRISPR to correct genetic mutations in cultured cells from Duchenne patients, and other labs had corrected genes in single - cell embryos in a laboratory environment.

The result was the largest deletion ever observed in the dystrophin gene using CRISPR / Cas9, and the study was the first to create corrected human iPS cells that could directly restore functional muscle tissue affected by Duchenne.

«We took patient - derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells.

«This work demonstrates the feasibility of using a single gene editing platform, plus the regenerative power of stem cells to correct genetic mutations and restore dystrophin production for 60 percent of Duchenne patients,» said Pyle, associate professor of microbiology, immunology and molecular genetics and member of the Broad Stem Cell Research Center.

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct a mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.

«I'm not sure if we will ever cure Duchenne muscular dystrophy, but I'm very hopeful that someday in the future, we will have new therapies that correct the ability of muscle stem cells to repair the muscles of afflicted patients and turn this devastating, lethal disease into a chronic but manageable condition.»

Scientists have developed a CRISPR gene - editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.

Some muscles, including the heart, were corrected by the therapy, and this is considered a major success because heart failure surfaces as the common cause of death among Duchenne sufferers.

The authors were able to correct mutations in several well - characterized genetic disorders, including: Duchenne Muscular Dystrophy, Achondroplasia, and MECP2 - duplication syndrome using cells derived from human patients.

Could a new CRISPR gene - editing technique correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy?