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Recent advances in CRISPR/Cas9 gene-editing tools, which the blog has highlighted in the past, have renewed hope that it might be possible to cure sickle cell disease by correcting DNA typos in just the right set of cells. Now, in a study published in Science Translational Medicine, an NIH-funded research team has taken an encouraging step toward this goal. For the first time, the scientists showed that it’s possible to correct the hemoglobin mutation in blood-forming human stem cells, taken directly from donors, at a frequency that might be sufficient to help patients.
news & eventsGlobal Blood Therapeutics Announces Completion of Safety Review by Independent Data and Safety Monitoring Board (DSM...Global Blood Therapeutics, Inc. (GBT) (N...
news & eventsWhat’s Inside My Medicine Cabinet?Living with sickle cell disease, I rely ...
news & eventsGBT expands sickle cell disease pipeline with worldwide licensing agreement for inclacumab for the treatment of vaso...Global Blood Therapeutics, Inc...
education & researchAllogeneic stem cell transplantation for sickle cell diseasePURPOSE OF REVIEW: As the safety and ava...
education & researchHow Is Sickle Cell Disease Treated?Babies with sickle cell disease (SCD) sh...
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videos & visualsSickle Cell Patients are Innocent Victims of the Opioid Epidemic: Samir K. Ballas, MDhttps://www.youtube.com/watch?v=BOhY_gJo...
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