Researchers from Oxford in Great Britain and Harvard in the United States have received a grant of 375 million SEK to develop a method to use the genetic scissor Crispr to cure inherited heart diseases.
This is a disease that often affects young people. In the UK alone, twelve people under the age of 35 die every week due to genetic defects that knock out the person's heart muscles.
With Crispr, the researchers should be able to correct exactly the genes that cause the problems and thus give those affected the opportunity to live a full life without any symptoms of heart problems.
The research team has already shown that the method works on human cells in the laboratory, so what they need to do now is to develop the method and move on to clinical testing.
What the researchers do is they first identify a faulty gene that produces an abnormal protein that damages the heart muscle. By turning off that gene, that damage is stopped.
The researchers can also correct a gene that does not produce enough of a certain protein that the heart muscle needs to function properly. Here, Crispr can be used to stimulate the gene to produce more of the protein.
The researchers expect to be able to start clinical testing within five years.
