muscular dystrophy

For several reasons, scientists, as well as gene editing therapy company Editas, think that the first clinical trials for CRISPR-based cures will be for hereditary eye diseases.

Researchers found that after viral delivery into muscle tissue or blood, CRISPR/Cas9 was able to excise disease-associated exons from the dystrophin gene.

Removing exon duplications is just one way in which the genome editing technology could be applied to treat hereditary genetic diseases.

This Week in Cell

In Cell this week: exploration of Yersinia pestis origins, modifier for Duchenne muscular dystrophy, and more.

Investigators from SomaLogic and elsewhere found proteins whose expression levels vary in Duchenne muscular dystrophy, often in conjunction with patients' age.

NEW YORK (GenomeWeb) – A consortium of limb-girdle muscular dystrophy (LGMD) family foundations has launched a program to provide free genetic testing to people suffering from muscular dystrophy.

NEW YORK (GenomeWeb) – Using the CRISPR/Cas9 genome editing system, researchers from the University of Texas Southwestern Medical Center were able to prevent Duchenne muscular dystrophy in a mouse model of the disease.

NEW YORK (GenomeWeb News) – Cellular Dynamics will create five induced pluripotent stem cell lines from patients with muscular dystrophy for the Jain Foundation under a master service agreement announced today.

NEW YORK (GenomeWeb News) – The Centre of Excellence for the Prevention of Organ Failure (PROOF Centre) and MRM Proteomics said today that they are collaborating with the Jain Foundation on research into biomarkers for muscular dystrophy.

NEW YORK (GenomeWeb News) – Drug firm Summit and the Children's National Medical Center in Washington today announced a collaboration for the development of a proteomic assay to measure biomarkers in support of Summit's candidate drug for Duchenne Muscular Dystrophy (DMD).

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New results from the NASA Twins Study indicate that the immune system may rev up when in space, the Washington Post reports.

Kelvin Droegemeier, the new science advisor, spoke at the American Association for the Advancement of Science meeting, Geekwire reports.

In PNAS this week: whole-genome assembly for the white shark, paper-based microfluidic method for detecting the malaria parasite, and more.

The World Health Organization has announced the members of its gene-editing committee, according to NPR.