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arginine + hydroxyurea
- Development stage
- Phase 1
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
A combination of arginine and hydroxyurea being investigated for treatment of sickle cell disease. Hydroxyurea increases fetal hemoglobin (HbF) synthesis through a nitric oxide (NO)-cGMP pathway, while arginine serves as the main precursor of nitric oxide. The combination aims to enhance HbF production and reduce sickle cell formation more effectively than hydroxyurea alone. Laboratory studies have shown that specific combinations of hydroxyurea with arginine can increase HbF synthesis while minimizing cytotoxicity compared to hydroxyurea alone.
02
Targets
NOS1 (nNOS)RNR (Ribonucleotide reductase)HbF (Fetal hemoglobin)NO2- (Nitrite)
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