Unmet Need / Invention Novelty: Expansions in nucleotide repeat elements have been linked to various neurological and neuromuscular disorders, with repeat-containing RNAs and/or their protein products being identified as important causes of pathogenic processes and associated diseases. There is an unmet clinical need to develop of a novel therapeutic strategy that can target nucleotide repeats to minimize and/or eliminate their products.
Technical Details: Johns Hopkins researchers have developed a novel strategy to decrease the toxic RNA and protein products generated from nucleotide repeats associated with neurological diseases such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The strategy consists of a novel CRISPR-Cas-based method to target nucleotide repeats. Proof of concept studies demonstrated a decrease in toxic protein levels from a targeted nucleotide expansion repeat in amyotrophic lateral sclerosis (ALS) patient cells.
Value Proposition:
Looking for Partners to: Develop & commercialize as a novel therapeutic strategy for diseases where nucleotide expansions are implicated in pathogenesis
Stage of Development: Pre-clinical
Data Availability: in vivo
