Therapeutic Gene Silencing in the Nervous System with CRISPR-Cas13d

Amyotrophic lateral sclerosis (ALS) is a  fatal neurogenerative disorders that affect 5,000 people in the U.S. each year. Huntington’s disease (HD) is another progressive and fatal neurogenerative disorder that affects nearly 30,000 people in the U.S. Both of these neurodegenerative disorders are associated with abnormal protein accumulation, such as SOD1 and HTT. CRISPR-Cas13 targets the genes of interest (i.e., SOD1 and HTT) and ultimately reduces the gene expression that are related to ALS and HD. Previous CRISPR technologies often use the Cas9 enzyme, which presents multiple problems with ease of delivery, size, and potential genomic damage to cells. Cas13 is smaller in size, making it easier to deliver. Furthermore, Cas13 targets the RNA—not the DNA, which means Cas13 downregulates gene expression without having to change the DNA sequence.