- Title: Scientists edit genome to cure sickle cell anemia
- Date: 10th November 2016
- Summary: BERKELEY, CALIFORNIA, UNITED STATES (RECENT) (REUTERS) VARIOUS OF RESEARCHER MARK DEWITT WORKING IN LAB SOUNDBITE (English) MARK DEWITT, POSTDOCTORAL FELLOW, UC BERKELEY, SAYING: "We know exactly the mutation that causes the disease and we know that if we correct that mutation we will cure the disease."
- Embargoed: 25th November 2016 16:09
- Keywords: sickle cell disease sickle cell anemia gene editing CRISPR-Cas9 UC Berkeley blood disorger genetic disease research science
- Location: BERKELEY, CALIFORNIA, UNITED STATES
- City: BERKELEY, CALIFORNIA, UNITED STATES
- Country: USA
- Topics: Life Sciences,Science
- Reuters ID: LVA00157V4B4R
- Aspect Ratio: 16:9
- Story Text:Using a gene editing technique called CRISPR-Cas9, researchers have corrected a genetic mutation that causes sickle cell disease, paving the way towards a potential cure.
"We know exactly the mutation that causes the disease and we know that if we correct that mutation we will cure the disease," Mark Dewitt of the University of California, Berkeley told Reuters.
Sickle cell disease is a painful genetic blood disorder that causes red blood cells to be abnormally crescent-shaped. These cells get stuck in blood vessels, blocking blood flow which leads to severe pain and organ damage.
The scientists focused their efforts on genetically flawed bone marrow stems cells that produce the abnormally shaped cells.
Using stem cell samples from people with sickle cell disease - they used CRISPR-Cas9 to edit out the mutation in the stem cells genome and replace it with a corrected version. They then implanted these edited cells into a mouse.
"We grafted the cells in the mouse for four months and after four months we took them out and we looked at the levels of editing and we were getting levels of editing consistent with achieving a clinical benefit for patients with sickle cell disease," said Dewitt. He added that the corrected stem cells produced healthy red blood cells efficiently enough to potentially cure the disease in human patients.
It will be some years before this research translates into treatment. The researchers need to scale up the process significantly, as well as prove its safety in humans.
The ability to edits genes is new science and the researchers need to prove that their efforts to cure one disease don't lead to others in the process.
But if proven harmless, gene editing could well be the answer to eliminating a host of genetic disorders in the years to come.
The findings were published last month in the online journal Science Translational Medicine.
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