Artistic rendering of genetic editing with medic using scissors to cut a slice of DNA
Artistic rendering of genetic editing with medic using scissors to cut a slice of DNA - A first-of-its-kind gene-editing therapy for people with a disorder that causes heart failure is safe in the short term and may stop disease progression, according to early results of a UCL-led trial. Transthyretin (ATTR) amyloidosis is characterised by a build up in the heart of misfolded transthyretin (TTR) protein. Symptoms include breathlessness, fatigue, and swelling in the legs. The condition gets progressively worse and is ultimately fatal within 2 to 10 years of diagnosis. Most of the treatment options available to patients before now have involved managing the symptoms rather than preventing progression of the disease. In a trial led by UCL researchers, a single injection of the novel gene-editing therapy NTLA-2001 was found to significantly reduce levels of circulating TTR protein by more than 90% after 28 days. Twelve patients with ATTR amyloid cardiomyopathy - that is, heart failure as a result of this condition - have received the treatment so far.
TO READ THIS ARTICLE, CREATE YOUR ACCOUNT
And extend your reading, free of charge and with no commitment.
