CRISPR has success in treating mice with type 1 diabetes

Insulin-producing cells have been restored in mouse models of type 1 diabetes using a new genetic engineering technique.
American scientists adapted the gene editing technology known as CRISPR (clustered, regularly interspaced, short palindromic repeat) to successfully treat mouse models of type 1 diabetes, kidney disease and muscular dystrophy.
CRISPR enables scientists to edit the genetic material of an organism allowing for DNA sequences to be easily altered and gene function to be modified.
The process builds on the established way of genetic engineering in a living being where malfunctioning genes are cut and removed or replacements are inserted. But the scientific community is excited by CRISPR because it speeds this technique up.
However, rather than cutting DNA, scientists at the Salk Institute for Biological Studies repurposed CRISPR so it kick-started the activity of other genes. Upon doing this, they were able to create new insulin-producing beta cells in mouse models of type 1 diabetes.
Explaining his team’s work, Professor Juan Carlos Izpisua Belmonte, Salk Institute for Biological Studies, said: “Cutting DNA opens the door to introducing new mutations. We are not fixing the gene; the mutation is still there.”
Because cutting DNA can result in “harmful mistakes”, Prof Belmonte said, using CRISPR to reverse the impact of diseases means there is no need for permanent genetic modification.
These findings are limited to mice, but researchers are buoyant it could provide hope for people with lifelong conditions such as type 1 diabetes. The research team is also hopeful it can offer potential to treat Alzheimer’s and Parkinson’s disease.
Dr Helen Claire O’Neill, a reproductive health specialist at University College London who was not involved in the study, said: “This paper clearly shows the potential therapeutic viability of this technology in human disease models.”
However, the study failed to explore any long-term results and impact on other parts of the body and Dr Alena Pance, a researcher at the Wellcome Trust Sanger Institute who was not involved in the study, said: “These issues would need to be investigated in depth before any application in humans is considered.”
The research has been published in the journal Cell.