Martin Roelsgaard Jakobsen
Targeting C9orf72 and neuroinflammation in ALS by novel dual directed CRISPR RNA therapy
Amyotrophic lateral sclerosis (ALS) is a rare and fatal disease, affecting approximately 1-3 individuals per 100,000. It is characterized by the progressive degeneration of nerve cells in the spinal cord and brain. The primary genetic culprit behind ALS is mutations in the gene called C9orf72, resulting in dysfunctional brain cells. The disease is exacerbated by the accumulation of toxic mutant C9 proteins and amplified neuroinflammation in the brain, accelerating the progression of ALS.
In this project, we aim to establish proof-of-concept for the precise delivery of innovative RNA therapeutics directly to nerve cells. These therapeutics are designed to reprogram nerve cells by suppressing the toxic mutant C9 protein, restoring healthy C9 protein and dampening neuroinflammation. Success in this endeavour will provide crucial pre-clinical feasibility data, paving the way for the development of novel disease-modifying therapies for ALS patients in the future.
