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takyon writes:

First step toward CRISPR cure of Lou Gehrig's disease

University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig's disease, in mice, extending their lifespan by 25 percent.

[...] The mice were genetically engineered to express a mutated human gene that in humans causes about 20 percent of all inherited forms of the disease and about 2 percent of all cases of ALS worldwide. Though the genetic cause is not known for all cases of ALS, all are accompanied by the premature death of motor neurons in the brain stem and spinal cord. The neurons allow the brain to control muscles, so loss of this connection means loss of muscle control.

[...] The UC Berkeley research team used a virus that Schaffer's team engineered to seek out only motor neurons in the spinal cord and deliver a gene encoding the Cas9 protein into the nucleus. There, the gene was translated into the Cas9 protein, a molecular scissors that cut and disabled the mutant gene responsible for ALS.

In this case, Cas9 was programmed to knock out the mutated gene SOD1 (superoxide dismutase 1). The onset or start of the disease was delayed by almost five weeks, and mice treated by the gene therapy lived about a month longer than the typical four-month lifespan of mice with ALS. Healthy mice can live a couple of years.

Lou Gehrig's disease = amyotrophic lateral sclerosis (ALS).

In vivo genome editing improves motor function and extends survival in a mouse model of ALS (open, DOI: 10.1126/sciadv.aar3952) (DX)

Previously: New Therapy Halts Progression of Lou Gehrig's Disease in Mice

Original Submission