NIH R01 · 2025
Targeted protein degraders for the treatment of b-hemoglobinopathies
Globally, 300,000 babies are born annually with Sickle Cell Disease (SCD). Current therapies for SCD revolve around induction of fetal hemoglobin (HbF), as it critically antagonizes red blood cell sickling. For example, hydroxyurea, a ribonucleotide reductase inhibitor, increases HbF levels. While hydroxyurea is the standard of care treatment for prevention of sickle cell crises, its effect on HbF induction can be weak and is variable from patient to patient. Attention has recently shifted to targeted gene therapy and gene editing efforts. For example, CASGEVY™ is a CRISPR-Cas9 gene editing technique to disable an erythroid specific enhancer of BCL11A to reactivate fetal hemoglobin gene…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.