After successful studies in mice, of a rare genetic disorder that affects children, UK scientists are ready to begin human trials of a new gene therapy.
The therapy, devised by researchers at EARA member the University of Manchester, and based on stem cells with the ability develop into any type of blood cell, will be trialled in five children under the age of one, with a condition known as Hunter syndrome.
Hunter syndrome arises from birth and affects the normal function of an enzyme called lysosome, that usually breaks down molecules such as fats and sugars, resulting in issues such as heart impairment and learning problems – in severe cases the life expectancy can be as little as 10 years.
It is hoped the new therapy will remove the need for weekly enzyme replacement therapy, as it is based on inserting a gene that will allow the body to create a working lysosome enzyme produced by the body’s blood cells.
Prof Brian Bigger, at Manchester, who led the research, said: “We’re very excited by the preclinical studies we carried out in mice, which showed the potential to correct disease in the body and normalise brain pathology.”