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Novel CRISPR-Cas based therapeutic strategy to decrease toxic RNA and protein products generated from nucleotide repeats

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...

Published: 3/14/2025 | Inventor(s): Jiou Wang, Honghe Liu

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Category(s): Technology Classifications > Therapeutic Modalities, Technology Classifications > Therapeutic Modalities > Gene Therapies