Gene therapy being developed at the University of Oulu to prevent the most common eye disease in premature infants

Retinopathy develops when the blood vessels in the retina of a prematurely born baby do not develop normally. The developmental disorder is promoted in particular by the high oxygen levels outside the womb. The greatest risk factors are birth before the 30th week of pregnancy, low birth weight and prolonged oxygen therapy.

Globally, retinopathy causes around 33,000 cases of severe visual impairment or blindness each year. Current treatments, retinal screening, laser therapy and drug injections, do not prevent the onset of the disease but address its consequences. The number of cases has increased as increasingly smaller premature infants survive thanks to modern intensive care.

Gene therapy aims at prevention

The research group led by Professor Peppi Karppinen at the University of Oulu is developing a treatment in which a single injection administered to the eye would prevent the development of retinopathy at an early stage. The method is based on long-term research into enzymes that regulate oxygen levels in the body. The gene therapy inhibits the action of a specific enzyme in the retina, allowing blood vessels to form normally.

Animal models have been used in the development work, and specific antibodies have been produced with the help of alpacas. The antibodies are delivered into cells via a virus, where they inhibit the enzyme’s activity. The gene therapy product is currently being prepared for preclinical trials, with the aim of progressing to clinical studies in the coming years.

The research project led by Karppinen is part of the GeneCellNano flagship programme funded by the Research Council of Finland.