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View Full Version : First sickle cell anemia "cure"



Angela
05-03-17, 19:20
Actually it's a reversal; what good news.

See:
http://www.bbc.com/news/health-39142971


"A French teenager's sickle cell disease has been reversed using a pioneering treatment to change his DNA.The world-first procedure at Necker Children's Hospital in Paris offers hope to millions of people with the blood disorder.
Scientists altered the genetic instructions in his bone marrow so it made healthy red blood cells."
"Doctors removed his bone marrow - the part of the body that makes blood. They then genetically altered it in a lab to compensate for the defect in his DNA that caused the disease.
Sickle cell is caused by a typo in the instructions for making the protein haemoglobin, which is densely packed into red blood cells.
A virus was used to infect the bone marrow with new, correct instructions.
The corrected bone marrow was then put back into the patient.
The results in the New England Journal of Medicine (http://www.nejm.org/doi/10.1056/NEJMoa1609677) showed the teenager has been making normal blood since the procedure 15 months ago."

LeBrok
05-03-17, 20:53
This is it, the medicine of 21st century! We fix problem where it starts, the DNA. Most devastating diseases could be ancient memory soon. Surprising is fact that this time the genetic cure came from France where DNA is a tabu. ;)