NIH R01 · 2025
Engineering human-derived programmable RNA effectors to retune gene expression
Project Summary Genetic deletion (haploinsufficiency) or duplication events are often associated with a variety of diseases, including: cancers, cardiovascular and metabolic disease, and neuropathies. Because many genes are sensitive to both over- and under-expression, with imbalances in gene product in either direction leading to disease, tight regulatory control is a therapeutic necessity. RNA-targeting technologies, however, provide a mechanism to appropriately and reversibly modulate gene expression at the transcript level, resulting in tunable, cell-specific remediation of disease-causing shifts in gene dosage. Our recently developed CRISPR- Cas- inspired RNA targeting system (CIRTS),…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.