Page URL:

Gene therapy temporarily halts type 1 diabetes in mice

8 January 2018
Appeared in BioNews 932

A gene therapy has been used to restore normal blood sugar levels in mice with type 1 diabetes, according to a study published this month in the journal Cell Stem Cell.

In type 1 diabetes the immune system destroys beta cells, but not alpha cells, in the pancreas. A team at the University of Pittsburgh School of Medicine and the Children's Hospital of Pittsburgh attempted to turn alpha cells into beta cells, in order to produce insulin. 

'If you gave patients new insulin cells with a transplant, it will kill them off. If we use gene therapy to get the body to make new insulin-producing cells in the body, logically it should attack those cells too,' explained Dr George Gittes, a lead study author and pediatric surgeon at the University of Pittsburgh, to Gizmodo.

But their treatment was successful in mice, at least temporarily. They used an adeno-associated virus, the standard gene therapy technology, to deliver two proteins, Pdx1 and MafA, to the pancreas of a mouse. This reprogrammed alpha cells into beta cells. Normal blood sugar levels were restored in the diabetic mice. However, this effect only lasted for around four months, though this would likely translate to years in humans.

'This study is essentially the first description of a clinically translatable, simple single intervention in autoimmune diabetes that leads to normal blood sugars,' said Dr Gittes. Additionally, many treatments targeting autoimmune disorders such as diabetes depress the immune system, causing lifelong risks. This treatment doesn't – a key advantage.

The gene therapy has only been tested on mice so far, but future work will be carried out in primates, and the group hopes to gain approval from the US Food and Drugs Administration (FDA) for clinical trials in humans. It is a promising new approach to tricking the immune system and alleviating the symptoms of diabetes. 

At present, type 1 diabetes has no cure, and is managed with a lifetime of insulin injections. Insulin is a hormone produced by pancreatic beta cells, which drives glucose absorption, lowering its levels in the body. 

The finding it is the latest of a string of promising developments in gene therapy. In October, the FDA approved Spark Therapeutics's gene therapy for a rare form of inherited blindness (see BioNews 922). The month also saw successful human trials on haemophilia A (see BioNews 931) and haemophilia B (see BioNews 930). 

Could Gene Therapy One Day Cure Diabetes?
GIZMODO |  4 January 2018
Endogenous Reprogramming of Alpha Cells into Beta Cells, Induced by Viral Gene Therapy, Reverses Autoimmune Diabetes
Cell Stem Cell |  4 January 2018
Gene therapy temporarily reverses diabetes in mice
FierceBiotech |  4 January 2018
Gene Therapy Temporarily Reverses Type 1 Diabetes in Mice
The Scientist |  4 January 2018
12 October 2020 - by Dr Joanne Delange 
A new extreme subtype of polygenic type 1 diabetes has been identified in infants under six months of age...
20 August 2018 - by Dr Kimberley Bryon-Dodd 
Scientists have partially restored sight in mice with congenital blindness by using gene therapy to activate retinal stem cells...
16 July 2018 - by Dr Rosie Morley 
Gene therapy has been used in mice to successfully reverse obesity and insulin resistance, as well as other markers of type 2 diabetes...
23 April 2018 - by Marcia Costa 
A clinical trial that used a new gene therapy to treat beta-thalassemia has shown very promising results...
5 March 2018 - by Shaoni Bhattacharya 
Scientists have coaxed human 'progenitor' cells from the pancreas to develop into the glucose-sensitive beta cells, and discovered their exact location within the organ...
11 December 2017 - by Isobel Steer 
Scientists in California have used a modified form of the CRISPR/Cas9 genome editing approach to epigenetically treat diabetes, kidney disease and muscular dystrophy in mice...
7 August 2017 - by Annabel Slater 
A proof-of-concept study in mice has demonstrated how skin grafts could deliver gene therapy for obesity and diabetes...
24 July 2017 - by Charlotte Spicer 
Genome-wide analysis in South Asian populations may provide insight into rare genetic diseases, suggests research...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.