Edit the genes slowed the development of amyotrophic lateral sclerosis in mice

Edit the genes slowed the development of amyotrophic lateral sclerosis in mice


Considered incurable degenerative disease called amyotrophic lateral sclerosis (also known as Lou Gehrig’s disease and Lou Gehrig’s disease) managed to slow down by editing genes in laboratory mice. For the first time in people with this disease among which the famous scientist Stephen Hawking, appeared tiny, but still hope that one day there will be a “magic pill” that can change their lives for the better. Because technology is CRISPR/Cas9 every year medicine opens up new opportunities that previously seemed the stuff of science fiction.

Amyotrophic lateral sclerosis became widely known not only for Hawking, but after the famous viral campaign in support of awareness about the disease. Surely you remember how in 2014, the entire Internet is filled the Ice Bucket Challenge videos in which celebrities like VIN Diesel, Mark Zuckerberg, bill gates and Barack Obama had overturned on the bucket of ice water challenging others. All attendees were transferred to Fund research of the disease $ 10. In the end, the campaign helped raise more than $ 40 million, making it possible to further research this disease.

Researchers from the University of California in Berkeley published in the journal Science Advances the results of their experiments on laboratory mice with a mutant human gene SOD1, responsible for about 20% of cases of inherited amyotrophic lateral sclerosis. Edited using CRISPR/Cas9 genes were delivered to the motor neurons of the spinal cord of mice using AAV viruses are almost completely safe for people. Proteins made cuts in the right areas of the genome, after which the cells were embedded in these new spaces, free from the mutations of genes.

Subjected to gene editing lab mice lived an average of one month longer than the others, which is a very good result, given that the holders of the mutated gene did not live longer than 4 months. Symptoms of the disease BASS these mice developed significantly more slowly. Scientists themselves recognize that it is, of course, does not mean that they found a 100% cure of the disease. However, the technique of gene editing for the first time so brought them to victory over the disease.