A team of researchers led by the University of Cambridge and the Wellcome Trust Sanger Institute has developed an alternative to embryonic stem cell therapy that could correct a genetic mutation from a patient’s own skin cell, grow it into a healthy liver cell and use them to replace the damaged tissue.
The research, conducted in test tubes and on mice, are yet to be tested on humans but could lead to a new range of specialised cell treatments.
Scientists believe that the ability to mend defective genes in stem cells, which create every type of tissue in the body, could herald a new era of therapies for a wide range of genetic disorders.
The researchers targeted a gene that protects against inflammation in the liver by producing a certain protein.
In patients with a mutation in the gene, the protein becomes trapped in the liver, and leads to cirrhosis of the liver and lung emphysema.
Using “molecular scissors” the scientists were able to cut away the mutated section of DNA from a stem cell and replace it with a corrected version.
The stem cells went on to generate liver cells, which contained normal protein levels, indicating that the previously faulty gene was now working normally.
“Our systems leave behind no trace of the genetic manipulation, save for the gene correction,” the Telegraph quoted Prof Allan Bradley, director emeritus of the Wellcome Trust Sanger Institute, as saying.
“These are early steps but, if this technology can be taken into treatment, it will offer great possible benefits for patients.”
The finding appears in Nature.