Tested on organoids

Researchers from Trinity College Dublin and University College London (UCL), combining their knowledge in the field of genetics, virology and ophthalmology, have developed a new treatment for retinitis pigmentosa.

Retinitis pigmentosa is a group of rare genetic disorders that are accompanied by the destruction and loss of photoreceptor cells in the retina. Most often, at an early stage, retinitis pigmentosa is accompanied by deterioration of twilight vision and loss of peripheral vision.

It is known that the disease develops due to a mutation in the RP2 gene, which is responsible for the expression of a protein necessary for normal vision. However, currently retinitis pigmentosa is not treated, patients irreversibly and almost completely lose their eyesight.

In the new study, the team used an adeno-associated virus to deliver a normally functioning copy of the RP2 gene to a "mini–retina" - a model that was constructed from stem cells and contained a defective version of the gene.

Subsequent analysis showed that in the experiment, the mini-retinas successfully captured the functioning RP2 gene and began to produce the protein that it encodes. In other words, the therapy cured the models of retinitis pigmentosa.

The authors write that they are aware of the amount of work required to approve the therapy. They also note that the developed approach may be useful for the study and treatment of other hereditary diseases of the retina.

Article A.Lane et al. Modeling and Rescue of RP2 Retinitis Pigmentosa Using iPSC-Derived Retinal Organoids is published in the journal Stem Cell Reports.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on the materials of Trinity College Dublin: Scientists rescue mini retinas from eye disease via new gene therapy approach.