End in sight for sickle-cell disease

09 November 2016 - 08:50 By Reuters

Scientists at Stanford University School of Medicine have used a gene editing tool to repair the gene that causes sickle-cell disease in stem cells taken from diseased patients - paving the way for a cure for the disease, which affects up to 5million people globally. "We've finally shown that we can do it," said Matthew Porteus, senior author of the report on the study, published in the journal Nature.With the study, and unpublished findings from his lab, Porteus believes his team has amassed enough proof to start planning the first human clinical trial using the powerful CRISPR-Cas9 gene editing system to correct the genetic mutation that causes sickle-cell disease.CRISPR works like molecular scissors, trimming away unwanted parts of the genome, and replacing it with new stretches of DNA.In sickle-cell disease, the body makes mutant, sickle-shaped haemoglobin, the protein in red blood cells that carries oxygen to the body's tissues. It is caused by a single mutation in a gene that makes a haemoglobin protein.The patient's own corrected stem cells would be injected to graft onto the bone marrow and produce healthy blood. ..

There’s never been a more important time to support independent media.

From World War 1 to present-day cosmopolitan South Africa and beyond, the Sunday Times has been a pillar in covering the stories that matter to you.

For just R80 you can become a premium member (digital access) and support a publication that has played an important political and social role in South Africa for over a century of Sundays. You can cancel anytime.

Already subscribed? Sign in below.



Questions or problems? Email helpdesk@timeslive.co.za or call 0860 52 52 00.