Monday 25 August 2014

» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne

» Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy Action Duchenne: "Sarepta Enters into Partnership with Flagship Biosciences to Digitally Automate the Measurement of Dystrophin, a Key Therapeutic Efficacy Marker for Muscular Dystrophy"



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New gene therapy protects mice from life-threatening heart condition

New gene therapy protects mice from life-threatening heart condition: "A new gene therapy developed by researchers at the University of Missouri School of Medicine has been shown to protect mice from a life-threatening heart condition caused by muscular dystrophy.

"This is a new therapeutic avenue," said Yi Lai, Ph.D., the leading author of the study and assistant research professor in the MU School of Medicine's Department of Molecular Microbiology and Immunology. "This is just a first step, but we hope this could lead to a treatment for people with this devastating heart condition, which is a leading cause of death for people with Duchenne muscular dystrophy.""



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Tuesday 19 August 2014

Researchers use new gene editing method to correct mutation that leads to DMD

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

Researchers used a technique called CRISPR/Cas9-mediated genome editing, which can precisely remove a mutation in DNA, allowing the body's DNA repair mechanisms to replace it with a normal copy of the gene. The benefit of this over other gene therapy techniques is that it can permanently correct the "defect" in a gene rather than just transiently adding a "functional" one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology."



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