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The funding received by researchers from Belgorod National Research University (BelSU) is part of the Federal Scientific and Technical Program for the Development of Genetic Technologies.

The research, led by scientists at BelSU Research Institute of Pharmacology of Living Systems in collaboration with the industrial partner Artgen Biotech Group, aims to develop ground-breaking technologies for genetic modelling and gene therapy targeting neurodegenerative diseases such as Alzheimer’s, Parkinson’s, and amyotrophic lateral sclerosis (ALS).

Central to the project is the investigation of the endogenous tetrapeptide HAEE, whose deficiency is linked to neurodegeneration. The team plans to create genetically modified animal models to identify the precursor protein of HAEE and to test new drugs utilizing adeno-associated viral vectors (AAV) to deliver neurotrophic factors aimed at restoring neuronal function.

Artgen Biotech’s involvement focuses on the commercialization of these technologies, the development of a platform for AAV therapy, and preparing necessary documentation for drug registration.

BelSU plays a crucial role in this initiative by providing the essential infrastructure and overseeing the implementation of the research program. The project will engage various research teams, particularly those specializing in gene editing, a bioresource collection of genetically modified animals, and a preclinical research centre. The university coordinates interdisciplinary collaboration among molecular biologists, pharmacologists, and neurobiologists. Leading the experimental research is Professor Mikhail Pokrovsky, Director of the Research Institute of Pharmacology of Living Systems, who will oversee disease modelling in animals and assess the effectiveness of therapies through behavioural tests and histological analyses. Molecular biology expert Alexey Deykin will spearhead efforts in creating and validating genetically modified animals through gene editing techniques. Moreover, Sergey Kozin, a patent author for HAEE, will explore the role of the endogenous tetrapeptide in amyloidosis pathogenesis and devise strategies to block toxic beta-amyloid aggregates. The overall research program is directed by Professor Mikhail Korokin, Deputy Director of the Research Institute.