Menu

CRISPR Improves Disease in Adult Mice

Three groups of researchers used the gene-editing method to restore a protein deficient in Duchenne muscular dystrophy.

Jan 4, 2016
Kerry Grens

WIKIMEDIA, CENTERS FOR DISEASE CONTROL AND PREVENTION'S PUBLIC HEALTH IMAGE LIBRARYCRISPR has fixed the protein problems in adult mice that lie at the root of Duchenne muscular dystrophy (DMD), a progressive disease that saps kids of muscle strength and ultimately shortens their lives. Scientists had succeeded in using the gene-editing technique to restore protein function in human cells or mouse embryos, but this is the first  time adult animals have been treated.

“The hope for gene editing is that if we do this right, we will only need to do one treatment,” Duke University’s Charlie Gersbach, who led one of three independent research teams that published results in Science last week (December 31), told The New York Times. “This method, if proven safe, could be applied to patients in the foreseeable future.”

The problematic protein is called dystrophin. All three groups took the same approach, first demonstrated in mouse embryos by Eric Olson of the University of Texas Southwestern Medical Center in 2014, to correct dystrophin deficiencies. They clipped a mutant exon from the gene for dystrophin, resulting in a truncated but functional protein. “Importantly, in principle, the same strategy can be applied to numerous types of mutations within the human DMD patients,” Olson, whose group was one of the three reporting successful results, said in a press release.

The researchers used viral vectors to deliver their genetic payload to tissues. The third team, led by Harvard University’s Amy Wagers, targeted muscle stem cells and heart muscle cells. The other teams injected the CRISPR-loaded vector into leg muscle or the bloodstream. Each approach succeeded to varying degrees, although as Science pointed out, none of the mice were fully cured. “There’s a ton of room for optimization of these approaches,” Gersbach told Science.

September 2018

The Muscle Issue

The dynamic tissue reveals its secrets

Marketplace

Sponsored Product Updates

Horizon Discovery introduces Myeloid DNA Reference Standard to support genetic testing of leukemia

Horizon Discovery introduces Myeloid DNA Reference Standard to support genetic testing of leukemia

Horizon Discovery Group plc, a global leader in gene editing and gene modulation technologies, today announced the launch of its Myeloid DNA Reference Standard. The first-to-market large cell-line derived myeloid cancer reference standard designed enables faster, more reliable and more cost-effective assay validation, to support the market in bringing routine testing into practice.

StemExpress LeukopakâNow Available in Frozen Format

StemExpress LeukopakâNow Available in Frozen Format

StemExpress, a Folsom, California based leading supplier of human biospecimens, announces the release of frozen Peripheral Blood Leukopaks. Leukopaks provide an enriched source of peripheral blood mononuclear cells (PBMCs) with low granulocyte and red blood cells that can be used in a variety of downstream cell-based applications.

New Antifade Mounting Media from Vector Laboratories Enhances Immunofluorescence Applications

New Antifade Mounting Media from Vector Laboratories Enhances Immunofluorescence Applications

Vector Laboratories, a leader in the development and manufacture of labeling and detection reagents for biomedical research, introduces VECTASHIELD® Vibrance™ – antifade mounting media that delivers significant improvements to the immunofluorescence workflow.

Best Practices for Sample Preparation and Lipid Extraction from Various Samples

Best Practices for Sample Preparation and Lipid Extraction from Various Samples

Download this white paper from Bertin Technologies to learn how to extract and analyze lipid samples from various models!