Scientists have developed a new type of genetic engineering, which they say has the potential to transform the way diseases are treated, and could be used to reverse the symptoms of some incurable conditions. The new technique removes a lot of the risks associated with gene modifying technology, and could bring a new and effective treatment for patients who currently require lifelong care.
The new tool, which is known as “Crispr” has been trialed on mice, and has successfully treated diabetes, kidney disease and muscular dystrophy. Researchers say that it could also be applied to a range of other untreatable conditions including Alzheimer’s and Parkinson’s disease.
The majority of genetic engineering techniques currently involve making cuts in malfunctioning genes, then removing or replacing them. The research into Crispr indicates that instead of cutting the genes, it can be used to kick start activity in order to reserve some of the effects of diseases.
Professor Juan Carlos Izpisua Belmonte from The Salk Institute for Biological Studies said that “cutting DNA opens the door to introducing new mutations”, adding that this breakthrough is a “major bottleneck in the field of genetics” which will help avoid the possibility of “harmful mistakes” being made from cutting DNA.
The new technique means there’s no need for permanent modification of genes, which can avoid harmful changes to DNA and remove a lot of risk for patients. Researcher Professor Belmonte and his colleagues used the modified Crispr tool to promote the activity of genes, which gave promising results for the treatment of diseases in mice.
The tool was tested in mice with diabetes to induce the production of insulin producing cells with positive results. Positive results were seen in mice with kidney disease, and in mice with muscular dystrophy researchers found they were able to recover healthy muscle function and growth. In diabetic mice, the tool induced the formation of cells that produce insulin – the hormone that is deficient in people with Type 1 diabetes. Similarly positive results were seen in the treatment of kidney disease.
Prof Belmonte noted that this technique isn’t like traditional gene editing tools, saying that “We are not fixing the gene; the mutation is still there.” The technique works by changing the way chemical interact with the gene and change its functionality. This can allow other genes to activate and recover the function of nonfunctioning DNA.
Dr Alena Pance, a researcher at the Wellcome Trust Sanger Institute commented that “These results bring hope for a targeted gene therapy and widens the application of the technology.” Dr Helen Claire O’Neill, a reproductive health specialist at University College London added that “This paper clearly shows the potential therapeutic viability of this technology in human disease models.”