Biotech company Editas Medicine is planning to start human trials to genetically edit genes and reverse blindness.
Humans who have had their DNA genetically modified could exist within two years after a private biotech company announced plans to start the first trials into a ground-breaking new technique.
Editas Medicine, which is based in the US, said it plans to become the first lab in the world to ‘genetically edit’ the DNA of patients suffering from a genetic condition – in this case the blinding disorder ‘leber congenital amaurosis’.
The disorder prevents normal function of the retina; the light-sensitive layer of cells at the back of the eye. It appears at birth or in the first months of life and eventually sufferers can go completely blind.
The rare inherited disease is caused by defects in a gene which instructs the creation of a protein that is essential to vision.
But scientists at Editas Medicine in the US believe they can fix the mutated DNA using the ground-breaking gene-editing technology Crispr.
Katrine Bosley, the chief executive of Editas Medicine, told a conference in the US that the company hopes to start trialling the technology on blind patients in 2017.
It would be the first time the technology has been used on humans. Gene editing is currently banned in the US, so the company would need special permission from health regulators.
“It feels fast, but we are going at the pace science allows,” Bosley told the EmTech conference in Cambridge, Massachusetts.
Crispr, which stands for Clustered, Regularly Interspaced, Short Palindromic Repeat, is a naturally-occurring defence mechanism used by bacteria.
Bacteria carry in their DNA strands of genetic code belonging to viruses so that they can recognise them when they come near. When they spot a virus they release an enzyme which attacks, snipping away this area of code.
Scientists have harnessed this mechanism to use as a kind of ‘molecular scissors’ which removes mutated areas of DNA.