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Gene therapy lets teen dodge sickle cell disease and properly working red blood cells to dodge the effects of the disorder

as mentioned in (CBS News) – A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report.
“It shows gene therapy is on the right track.”
Gene therapy offers hope of a lasting one.
About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found.
Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen.

Gene therapy lets teen dodge sickle cell disease

as declared in Hospital group Sanford Health has invested $20 million in InGeneron to support clinical trials of a stem cell regenerative medicine.
The Series D gives Sanford a financial stake in an adipose-derived stem cell therapy it is testing in a clinical trial at its network of healthcare facilities.
InGeneron began working with Sanford on an 18-person trial of its stem cell injection in patients with partial thickness rotator cuff tears around the turn of the year.
“This significant investment demonstrates Sanford’s commitment to be an active participant in InGeneron as well as being our clinical trial site of choice.
That financing, which came 10 years after InGeneron was founded, followed studies of the company’s cell therapies in knee surgeries.
Hospital group invests $20M in stem cell therapy biotech

as declared in Sickle cell disease causes normally round red blood cells, which carry oxygen around the body, to become shaped like a sickle.
But the study does show the potential power of gene therapy to transform the lives of people with sickle cell.
Image copyright SPL Image caption Healthy red blood cells are round, but the genetic defect makes them sickle shapedA French teenager’s sickle cell disease has been reversed using a pioneering treatment to change his DNA.
Image copyright Science Photo LibraryWhat is sickle cell disease?
However, the expensive procedure can only be carried out in cutting-edge hospitals and laboratories, while most sickle cell patients are in Africa.

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