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CRISPR Hopes for ALS

Researchers are testing whether gene editing might be possible treatment avenue for diseases like amyotrophic lateral sclerosis, Gizmodo reports.

While most cases of ALS aren't familial, a small portion is, and about 20 percent of those familial ALS cases are due to mutations in the SOD1 gene. A University of California, Berkeley-led team of researchers used the CRISPR-Cas9 gene editing machinery to knock out the mutated gene in a mouse model of autosomal dominant ALS, as the Scan also notes here. As the researchers report in Science Advances this week, treated mice had reduced levels of mutant SOD1 protein in their lumbar and thoracic spinal cord, which led to better motor function and reduced muscle atrophy. Gizmodo notes that the mice weren't cured, but that they did live longer and had delayed disease onset.

Berkeley's David Schaffer and his colleagues write in their paper that their findings suggest that gene editing could be developed to treat central nervous system disorders that are due to autosomal dominant mutations. "I've been in gene therapy for 25 years, but only the advent of CRISPR has raised the possibility of erasing a gene," Schaffer tells Gizmodo. "That's where ALS comes in."

However, Schaffer notes that there's still the issue of delivery to contend with.

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