Italian scientists have found a way to tweak a gene in mice to stop a potentially lethal blood condition called hemophilia developing.
It is the first time that gene therapy has been used to treat blood diseases.
Doctors and researchers at Milan's prestigious San Raffaele Hospital took fragments of genetic material that regulate the expression of genes, called micro-RNA, and modified them to fool the body's immune system.
"We worked out a strategy to hide the new gene from the immune system and we successfully applied it to the hemophilia model in mice," said the head of San Raffaele's TIGET gene therapy lab, Luigi Nardini.
Nardini warned that it would take some time to apply the breakthrough to humans.
"First we will have to show that it is safe and effective in larger animals," said Nardini, whose work is set for publication in major international journals.
"The path towards clinical testing is rather long but the approach via micro-RNA appears to be very promising," Nardini said.
Hemophilia, a rare bleeding disorder that prevents the blood from clotting properly, is a nightmare when it comes to the bumps and scrapes of every child's life.
About one in every 8,000 boys is born with hemophilia, while girls are more rarely affected because it is linked to gender.
