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3 Gene Editing Approaches for Sickle Cell Disease
Sickle cell disease (SCD) is a perfect candidate for gene editing. It is perhaps the best understood single-gene condition, due to a substitution of a single DNA base in the gene that encodes the beta subunit of hemoglobin, the protein that carries oxygen in the blood.
Red blood cells, which bend into the sickle shape in the disease, obstructing circulation and causing excruciating pain, descend from hematopoietic (“blood-forming”) stem cells (HSCs) in the bone marrow. So theoretically, a patient’s stem cells can be removed, the mutation replaced in them, and then the fixed cells infused back into the patient, circumventing an immune response.
education & researchFrom total blood exchange to erythrocytapheresis and back to treat complications of sickle cell diseaseErythrocytapheresis is an important proc...
news & eventsASH launches sickle cell disease clinical trials network to accelerate therapy developmentASH launched a clinical trials network d...
news & events100 Years But Only One Drug: Sickle Cell Patients Wait For HelpLast week, 100,000 Americans with sickle...
education & researchSickle Cell Disease: Managing PainCurrently, sickle cell disease has no wi...
news & eventsSickle Cell Disease Research Shows Progress in Preventing Related Complications and DeathStudies aim to limit pain crises, preven...
Community CenterSuccessful Gene Therapy for Sickle Cell DiseasePrior to this report, the only curative ...
news & eventsGhana Launches Partnership With Novartis to Improve Diagnosis and Treatment of People With Sickle Cell DiseaseThe Government of Ghana announced a new ...
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