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- Researchers develop first CRISPR medicine for sickle cell disease
Five researchers from Harvard and NIH received the 2026 Warren Alpert Foundation prize for identifying BCL11A as a key regulator of the fetal-to-adult hemoglobin switch, enabling the development of Casgevy, the first CRISPR medicine for sickle cell disease and thalassemia.
- Field
- gene therapy
- What they did
- Scientists from Harvard and NIH received an award for discovering the role of the BCL11A protein in switching from fetal to adult hemoglobin, which became the basis for developing the first CRISPR-based medicine for sickle cell disease and thalassemia.
- Why it matters
- This discovery enabled the development of therapy that can correct genetic defects causing these diseases using genome editing technology.
#crispr#hemoglobin#bcl11a#sickle cell#gene editing
