This step toward a CRISPR cure for human ALS will be reported Dec. 20 in the journal Science Advances.
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 devastating disease usually strikes people between the ages of 40 and 70.
There, the gene was translated into the Cas9 protein, a molecular scissors that cut and disabled the mutant gene responsible for ALS.
One of several challenges is to eliminate the SOD1 mutation in other brain and spinal cord cells that support motor neurons.
UC Berkeley co-authors of the paper are Thomas Gaj, David Ojala, Freja Ekman, Leah Byrne and Prajit Limsirichai. »