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mRNA Intensifier to treat Haploinsufficiencies

Unmet Need / Invention Novelty: Haploinsufficiency occurs when one gene allele is inactivated and the amount of gene product expressed from the remaining active allele is insufficient for proper gene function. Targeted restoration of mRNA expression and translation may offer a therapeutic window. Technical Details: Johns Hopkins researchers designed...

Published: 3/14/2025 | Inventor(s): Jeffery Coller, Bahareh Torkzaban

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