CRISPR/Cas9 Based Preclinical Advances for Amyotrophic Lateral Sclerosis

What is ALS?

Amyotrophic lateral sclerosis (ALS), originally known as Lou Gehrig’s disease, is a rare neurological disorder leading to motor neuron degeneration and ultimately cell-death. ALS is fatal and characterized by a rapid degeneration process, which leads to paralysis and typically culminates in respiratory failure within 3-5 years following the onset of symptoms (Amado and Davidson 2021).

Globally, the prevalence of ALS ranges between 4.1 - 8.4/100,000 people. In the US, various population-based studies support a prevalence change from 3.7/100,000 in 2002 to 5.2/100,000 in 2015. Ethnicity, specifically of European descent, is linked to greater disease prevalence (Longinetti and Fang 2019).

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