Neurosoft in Scientific Collaboration on Gene Therapy Development for Rare Hereditary Diseases

Recently, representatives of Neurosoft’s Non-invasive Neuromodulation team visited one of the branches of the Federal Research Center for Original and Advanced Biomedical and Pharmaceutical Technologies, where gene therapies based on adeno-associated vectors are being developed for the treatment of rare hereditary diseases.

One of the research groups is working on the problem of adrenoleukodystrophy. Their ultimate goal is to stop neurodegeneration and prevent a fatal outcome. To achieve this, the scientists are creating a mouse model whose phenotype closely reproduces the human one. The study will last approximately one year, and the animals will remain in the experiment throughout its duration. At several predefined checkpoints (up to 40 mice at each), motor, behavioral and electrophysiological tests will be performed. This will make it possible to demonstrate the effectiveness of the therapy not only at the molecular level but also at the functional level.

Particular importance in this work is given to the objective assessment of the neuromuscular system and corticospinal tract conduction. For the study, the project team uses the Neurosoft Transcranial Magnetic Stimulation system equipped with a coil for translational research on small animals and a dedicated cooling unit and the Litebox electroneuromyograph.

During the visit a comprehensive technical training session was conducted, and the operation of the software and equipment was demonstrated both on humans and on rodents. And of course it was a pleasure to meet the research team in person.

Our company provides the researchers with comprehensive methodological and technical support. We believe that behind every recorded mouse motor response lies the future of saving human lives.

Follow the progress of the project with Neurosoft!