NIH R01 · 2025
Chemical tools for modulating the fetal hemoglobin inducer BCL11A
Project Summary/Abstract Proximity-based protein modulators such as proteolysis targeting chimeras (PROTACs) and molecular glues represent a new therapeutic modality because they can, through proximity-induced protein depletion, ablate all functions of their targets. Because of their ability to target non-enzymatic proteins, these molecules have been heralded as key to eliminating the “undruggable” human proteome. However, the transformational potential of this technology is limited by the necessity of drug-like ligands for target proteins, which are sometimes intrinsically disordered or do not possess small molecule binding pockets. One of these target proteins is BCL11A, the…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.