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The Major Advancement in Sickle Cell Treatment That Has Everyone Talking

November 18, 2016 by Andrea
Photo: Public Domain

Photo: Public Domain



Over the past week, there have been some exciting developments toward the treatment of sickle cell. One has to do with Xickle's very own journey from supplement status to pharmaceutical drug status (more on this in the coming weeks). Another has to do with the developments in gene editing from scientists at Standford University School of Medicine. 

At Stanford, those scientists have successfully used the gene-editing tool, CRISPR, on the very gene that causes sickle cell -- mending it in stem cells taken from sickle cell warriors. The study, which was published last week in Nature, discusses how Dr. Natthew Porteus and his team repaired the gene mutation in 30 to 50 percent of cells with SCD. Then, those cells were injected into mice, and according to Reuters, those same cells were still functioning properly 16 weeks later.

"What we've finally shown is that we can do it," said Porteus in ScienceDaily. "It's not just on the chalkboard. We can take stem cells from a patient and correct the mutation and show that those stem cells turn into red blood cells that no longer make sickled hemoglobin."

The next step? Human clinical trials. Right now, the plan is to begin those trials in 2018. This is definitely a historic advancement in the treatment of SCD, and we're excited to see where it leads.

For more information on the study and the process planned for human clinical trials, read the entire articles below:

1) ScienceDaily: "Step toward gene therapy for sickle cell disease"
2) Reuters: "Stanford uses CRISPR to correct sickle cell, human trials planned"
3) TheScientist: "More Success Fixing Sickle Cell Gene with CRISPR"


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