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Protein Restoration

Researchers led by Kyoto University's Shinya Yamanaka compared the use of CRISPR- and TALEN-based approaches to edit dystrophin gene mutations in iPS cells derived from a Duchenne muscular dystrophy patient.

As they report in Cell Stem Cell, Yamanaka and his colleagues also tried three different strategies for modifying the loss-of-function dystrophin gene mutation: exon skipping, frameshifting by adding indel mutation, and knock-in of the missing exon. Exon knock-in, which restored full-length dystrophin protein, worked best, the researchers report.

Corrected iPS cells, they further report, could be differentiated into skeletal muscles. Using patient-derived iPS cells to treat Duchenne muscular dystrophy may limit rejection, though the researchers note that additional challenges, such as how to best transplant iPS cell-derived myogenic cells, need to be overcome before it can be tried clinically.

The researchers also report that using a k-mer database enabled them to find a unique target region and reduce off-target effects. 

While such an approach can't fix all the muscle fibers in a patient, the Guardian's Ian Sample notes that if enough muscle fibers can be targeted with a fix, it could still improve quality of life.

"It's a lovely approach. If these cells integrate into muscle in animals I'd expect it to work in humans because skeletal muscle is skeletal muscle," Chris Mason from University College London tells Sample. "You don't need to correct everything. If you can improve muscle strength by a small amount, you can help people in normal daily [activities]."

The Scan

Comfort of Home

The Guardian reports that AstraZeneca is to run more clinical trials from people's homes with the aim of increasing participant diversity.

Keep Under Control

Genetic technologies are among the tools suggested to manage invasive species and feral animals in Australia, Newsweek says.

Just Make It

The New York Times writes that there is increased interest in applying gene synthesis to even more applications.

Nucleic Acids Research Papers on OncoDB, mBodyMap, Genomicus

In Nucleic Acids Research this week: database to analyze large cancer datasets, human body microbe database, and more.