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Updated: Apr 7, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
2015 Clinical trials update in sickle cell anemia
Natasha Archer1, Frédéric Galacteros2, Carlo Brugnara3
1Pediatric Hematology/Oncology Dana-Farber/Children's Hospital Blood Disorders and Cancer Center, Boston, Massachusetts.
Sickle cell disease (SCD) involves HbS polymerization and red blood cell sickling. New therapies target these processes and associated inflammatory changes to prevent organ damage.
Area of Science:
- Hematology
- Molecular Biology
- Pathophysiology
Background:
- Sickle cell disease (SCD) is characterized by polymerization of hemoglobin S (HbS) and red blood cell sickling.
- A single amino acid substitution in the beta chain of hemoglobin drives HbS polymerization and altered red cell morphology.
- Oxygen tension and intracellular HbS concentration are key molecular drivers of HbS polymerization.
Purpose of the Study:
- To review the pathophysiology of SCD with a focus on therapeutic targets.
- To discuss molecular drivers and associated cellular changes in SCD.
- To outline current and future therapeutic interventions for SCD.
Main Methods:
- Review of molecular mechanisms of HbS polymerization.
- Analysis of cellular changes contributing to SCD pathophysiology.
- Examination of pre-clinical and clinical therapeutic developments.
Main Results:
- Understanding HbS polymerization and sickling has advanced significantly.
- Associated cellular changes contribute to inflammatory vasculopathy and organ damage.
- Numerous new therapeutic targets have emerged.
Conclusions:
- Targeting HbS polymerization and associated inflammatory pathways offers therapeutic potential.
- Further research into SCD pathophysiology is crucial for developing effective treatments.
- Multiple therapeutic strategies are under investigation for SCD management.
More Related Videos
08:23Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
Published on: November 5, 2019
05:23Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
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