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.

November 2018

Intelligent Science

Wrapping our heads around human smarts

Marketplace

Sponsored Product Updates

Complete Pathology Solutions: Make Every Minute Count

Complete Pathology Solutions: Make Every Minute Count

From sample collection and handling, to fixation and processing, tissue staining, and covering all your IHC and water purification needs—you can have confidence in the quality of your results with MilliporeSigma's one-stop pathology solution.

Preparing Cell Or Tissue Lysates For ELISA Kits

Preparing Cell Or Tissue Lysates For ELISA Kits

RayBiotech manufactures over 2,000 high fully validated, GMP-compliant ELISA kits. In this blog post we explain how to prepare cell or tissue lysates for ELISA Kits.

Norgen Biotek Achieves Illumina Propel Certification as a Service Provider for Next Generation Sequencing

Norgen Biotek Achieves Illumina Propel Certification as a Service Provider for Next Generation Sequencing

Norgen Biotek Corp., an innovative privately held Canadian biotechnology company focusing primarily on nucleic acid and protein stabilization and purification, as well as providing high quality services to the scientific community, today announced that it has become Propel-Certified through Illumina as a Next Generation Sequencing (NGS) service provider.

Slice® Safety Cutters for Lab Work

Slice® Safety Cutters for Lab Work

Slice cutting tools—which feature our patent-pending safety blades—meet many lab-specific requirements. Our scalpels and craft knives are well suited for delicate work, and our utility knives are good for general use.