Study doctors will use a ‘gene changing’ tool called CRISPR. The therapy changes the sickle gene mutation in a patient’s stem cells to a non-sickle gene that makes healthy hemoglobin. These gene-edited stem cells will be given back to the patient. 

Patients will undergo high-dose chemotherapy to make room for the gene-edited stem cells to grow. They are then transplanted back into the patient. 

This therapy has the potential to improve the health of individuals with sickle cell disease.

The post Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease first appeared on Sickle Cell Studies.

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The post Protected: The DREAM Clinical Trial first appeared on Sickle Cell Studies.

This study seeks to determine mitapivat’s safety and effectiveness versus placebo in study participants with sickle cell disease over a year. the effects it has on people with sickle cell disease who take the therapy and its safety over a longer period of 1 year compared to individuals who take a placebo living with sickle cell disease.

The post RISE UP: Studying whether an investigational drug can increase hemoglobin and reduce pain crises first appeared on Sickle Cell Studies.

The post Using Community Based Research Practices to Advance Anti-Racism in Sickle Cell Disease Clinical Care first appeared on Sickle Cell Studies.

The post Attributional Style and Adherence to Hydroxyurea in Adults with Sickle Cell Disease (HU Adherence Study) first appeared on Sickle Cell Studies.

• Significantly greater effort during exercise than normal
• Unusual muscle weakness and pain, commonly in the legs and lower back
• Normal to modest temperature elevation while initially responsive

The post Exercise Collapse Associated with Sickle Cell Trait (ECAST) first appeared on Sickle Cell Studies.

The post KNOWledge and Understanding of Sickle Cell Trait (KNOWUrSCT): A Global Health Disparity Research Study first appeared on Sickle Cell Studies.

NDec is an oral drug therapy being studied in sickle cell disease that could potentially improve hemoglobin. NDec is a combination of two medicines: decitabine and tetrahydrouridine.

The post ASCENT1 Study first appeared on Sickle Cell Studies.

The post An Electronic Patient Reported Pain Assessment in Sickle Cell first appeared on Sickle Cell Studies.

The post Hibiscus Study: An oral investigational medication for SCD first appeared on Sickle Cell Studies.