Programme “Towards precision medicine and gene therapy”

Gene therapy is a rapidly evolving field of biomedical and clinical research. Not only is the capacity for personalized sequencing increasing, which significantly improves the detection of new genetic diseases, but also the portfolio of gene editing and delivery tools, enabling precise correction of gene functions, is expanding. Along with the possible development of gene therapies in rare genetic diseases, these methods can be an enormous benefit for the treatment of diseases associated with aging or for regenerative medicine.

The Strategy AV21 of the Czech Academy of Sciences responds to current social challenges through a sophisticated formulation of research programmes, based on cooperation of scientific fields and institutions. In January 2022, a new research programmeTowards precision medicine and gene therapy” coordinated by the Institute of Molecular Genetics of the Czech Academy of Sciences was approved by the Science Council of the Czech Academy of Sciences.

The gene delivery via adeno-associated viruses (AAV) became a gold standard in the therapeutic gene delivery although it is still obscured by technological limitations and lack of complex preclinical research. However, there are still substantial limitations in advancing gene therapy towards patients, which are subject of investigation in the consortium of the Czech Academy of Sciences consisting of the Institute of Molecular Genetics, the Institute of Animal Physiology and Genetics, the Institute of Experimental Medicine and the Institute of Organic Chemistry and Biochemistry. Our overarching goal is to accelerate the transfer of experimental gene therapy approaches to clinical research and create a platform for the development, evaluation and standardization of the gene therapy processes.

The programme will also develop synergistic interactions with the AV21 programmes “Preclinical Testing of Potential Drugs” and “Virology and Anti-Virus Therapy” and opens opportunities for collaboration with pharmaceutical sphere. The programme will utilize state-of-the-art gene editing tools such as technologies CRISPR/Cas-e and inhibiting RNA and will further develop their use. Our programme benefits from the synergistic expertise of all partners and creates a multidisciplinary consortium covering the areas of gene editing, organic chemistry of nucleic acid delivery systems, preclinical models of human diseases, and starting with neurosciences will spread further to other physiological systems.

Our common effort will advance gene therapy for near future exploitation. We shall also disseminate this knowledge to Czech research communities and communicate the new opportunities to clinical institutes, patient organizations, pharma/biotech companies, and decision-making bodies to support development and implementation of new future therapies for patients.

1) to characterize new pathogenic gene variants that can serve as new gene therapy targets,
2) to provide proof-of-concept of gene therapy for specific diseases in vitro and in vivo models,
3) to develop new tools for gene/drug delivery.


Research Projects of the Members of the “Gene therapy” AV21 Consortium

New therapies for rare diseases of central nervous system and skin

Coordinator: Radislav Sedlacek

Czech Centre for Phenogenomics

Glia-oriented gene therapy in the disorders of the central nervous system

PI: Miroslav Anderova

Department of Cellular Neurophysiology

Potential therapeutic approaches to the treatment of Huntington’s disease

PI: Zdenka Ellederova

Laboratory of Cell Regeneration and Plasticity

Topically applied nanosystems for siRNA therapy

PI: Petr Cigler

Laboratory of Synthetic Nanochemistry