Gene-editing breakthrough – what does this mean for DC?

Photo credit: The Times


The Times reports that a pioneering gene-editing treatment is likely to have been successful in treating a little girl suffering from leukaemia. This follows an article last year, which first reported on the procedure.


Two-year old Layla could potentially be the first person in the world to have been saved by gene-editing, which she underwent after chemotherapy and a bone marrow transplant both failed to treat her cancer. Until her operation in June 2015, in which researchers implanted modified genes taken from a donor’s immune cells, gene-editing had only been carried out in a laboratory.


Layla has now been clear of leukaemia for long enough that her doctors at Great Ormond Street Hospital are now prepared to talk in terms of a potential cure.


Gene editing differs from conventional gene therapy in that it involves cutting away elements from the cell’s DNA using ‘molecular scissors’. In Layla’s case, this involved taking a donor’s healthy T-cells and modifying them using gene editing to create a bespoke treatment. Unlike conventional gene therapy, using a donor’s cells for gene editing does not require a perfect match.

Source: The Times


Although the process would likely be much more complex for someone with dyskeratosis congenita, this is an exciting breakthrough that could pave the way for future treatment of the condition, by correcting the genetic abnormality which causes it. Watch this space!


The full article is available to read here.