Menu

CRISPR Restores Muscle Function in Mice

Scientists use the gene-editing tool to treat animals with a rare form of congenital muscular dystrophy.

Jul 17, 2017
Diana Kwon

ISTOCK, DRA_SCHWARTZUsing CRISPR, researchers have successfully treated congenital muscular dystrophy type 1A (MDC1A), a rare disease that can lead to severe muscle wasting and paralysis, in mice. The team was able to restore muscle function by correcting a splicing site mutation that causes the disorder, according to a study published today (July 17) in Nature Medicine

“Instead of inserting the corrected piece of information, we used CRISPR to cut DNA in two strategic places,” study coauthor Dwi Kemaladewi, a research fellow at the Hospital for Sick Children (Sick Kids) in Toronto, explains in a statement. “This tricked the two ends of the gene to come back together and create a normal splice site.”

By targeting both the skeletal muscles and peripheral nerves, the team was able to improve the animals’ motor function and mobility. “This is important because the development of therapeutic strategies for muscular dystrophies have largely focused on improving the muscle conditions,” Kemaladewi says in the release. “Experts know the peripheral nerves are important, but the skeletal muscles have been perceived as the main culprit in MDC1A and have traditionally been the focus of treatment options.”

“The robustness of the correction we see in animal models to me is very encouraging,” Amy Wagers, a biologist at Harvard University who was not involved in this study, tells the Toronto Star.

See “CRISPR Improves Disease in Adult Mice

Wagers’s group and others have used CRISPR to fix a protein deficiency in adult mice with another rare muscle disease, Duchenne muscular dystrophy (DMD). Kemaladewi and colleagues have also tackled this disorder—in a 2015 study, their team used the gene editing tool to remove a duplicated gene and restore protein function in the cells of a patient with DMD.

See “CRISPR Therapy in a Dish

“For the first time it’s possible to think about—and this is still at the thinking stage, let’s be clear—the possibilities of gene correction in humans with these diseases,” Janet Rossant, a stem cell and developmental biologist at Sick Kids who was not involved in the research, tells the Toronto Star.

January 2019

Cannabis on Board

Research suggests ill effects of cannabinoids in the womb

Marketplace

Sponsored Product Updates

FORMULATRIX® digital PCR technology to be acquired by QIAGEN
FORMULATRIX® digital PCR technology to be acquired by QIAGEN
FORMULATRIX has announced that their digital PCR assets, including the CONSTELLATION® series of instruments, is being acquired by QIAGEN N.V. (NYSE: QGEN, Frankfurt Stock Exchange: QIA) for up to $260 million ($125 million upfront payment and $135 million of milestones).  QIAGEN has announced plans for a global launch in 2020 of a new series of digital PCR platforms that utilize the advanced dPCR technology developed by FORMULATRIX combined with QIAGEN’s expertise in assay development and automation.
Application of CRISPR/Cas to the Generation of Genetically Engineered Mice
Application of CRISPR/Cas to the Generation of Genetically Engineered Mice
With this application note from Taconic, learn about the power that the CRISPR/Cas system has to revolutionize the field of custom mouse model generation!
Translational Models of Obesity, Dysmetabolism, Diabetes, and Complications
Translational Models of Obesity, Dysmetabolism, Diabetes, and Complications
This webinar, from Crown Bioscience, presents a unique continuum of translational dysmetabolic platforms that more closely mimic human disease. Learn about using next-generation rodent and spontaneously diabetic non-human primate models to accurately model human-relevant disease progression and complications related to obesity and diabetes here!
BiochemAR: an augmented reality app for easy visualization of virtual 3D molecular models
BiochemAR: an augmented reality app for easy visualization of virtual 3D molecular models
Have you played Pokemon Go? Then you've used Augmented Reality (AR) technology! AR technology holds substantial promise and potential for providing a low-cost, easy to use digital platform for the manipulation of virtual 3D objects, including 3D models of biological macromolecules.