Zinc Finger Protein Technology Shows Promise as a Treatment for ALS

Amy Pooler, PhD, Vice President and Head of Neuroscience at Sangamo Therapeutics, discusses the company’s recent presentation on the use of zinc finger protein technology as a potential treatment for amyotrophic lateral sclerosis (ALS).

ALS, also known as Lou Gehrig’s disease, is a fatal neurodegenerative disease, characterized by the loss of motor neurons in the central nervous system, leading to paralysis and early death. The most frequent genetic cause of ALS is the expansion of hexanucleotide GGGGCC repeats in the first intron of the C9ORF72 gene. These repeats lead to sense and antisense transcriptions that are believed to be part of the pathophysiology of ALS.

As Dr. Pooler explains, current treatments available for ALS, as well as many other neurodegenerative disorders, primarily target symptoms of the disorder rather than the genetic cause. Sangamo hopes the use of zinc finger protein transcription factors (ZFP-TFs) can be used to target the GGGGCC repeats in ALS.

Recently, researchers at Sangamo presented data at the ASGCT 2021 meeting on use of ZFP-TFs to selectively repress C9ORF72 repeat expansion-containing sense and antisense transcripts as a potential treatment of ALS. Multiple ZFP-TFs were identified as capable of selectively repressing the expression of both ALS sense and antisense transcripts over a wide dose range while preserving the expression of healthy C9ORF72 mRNA levels.

Based on these encouraging results, pre-clinical studies are currently underway to evaluate ZFP-TFs in ALS mouse models.

To learn more about ALS and other rare neurological diseases, visit checkrare.com/diseases/neurology