This Project Grant award from the National Institute on Aging (CFDA 93.866 - Aging Research) provides $228,019 to Acrobat Genomics, Inc. to develop a high-throughput protein engineering platform to enhance their proprietary ARGN gene editing system for treating amyotrophic lateral sclerosis (ALS). The goal is to optimize the ARGN system to safely and efficiently deplete the disease-causing mutant C9ORF72 gene, which is a major genetic driver of ALS. The 22-month project will utilize advances in oligonucleotide synthesis, sequencing, and protein language models to engineer an improved ARGN variant capable of restoring expression of the wild-type C9ORF72 allele. This work aims to lay the foundation for a new gene therapy approach to this devastating and incurable neurodegenerative disease, for which there are currently limited treatment options.
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