gene therapy

This review focuses on the structure and molecular action mechanisms of chimeric antigen receptors (CARs) and major aspects of the manufacturing and clinical application of products for the CAR-T (CAR-modified T lymphocyte) therapy of hematological and solid tumors with special emphasis on the strategies for combined use of CAR-T therapy with immuno-oncological monoclonal antibodies (checkpoint inhibitors) and cytokines to boost survival, persistence, and antitumor efficacy of CAR-T therapy. The review also summarizes preclinical and clinical data on the additive effects of the combined use of CAR-T therapy with interleukins and monoclonal antibodies targeting immune checkpoints.

At the present time, gene technologies play a key part in the development of new approaches to the diagnostics and therapy of diseases. Russian scientists have already successfully introduced efficient preparations based on multiclonal antibodies into practice and have advanced developments in the fields of CAR-T therapy and gene-therapy preparations on the basis of recombinant adenoassociated viruses. Some gene diagnostic tools, which essentially improve the efficiency of medical care due to the personalized approach to the therapy of diseases, are also put into practice. The successful introduction of innovative technologies and contemporary methods of diagnostics and therapy is impossible without cooperation between academic science and business. The companies providing the conditions for cooperation with academic science and engagement of specialists of different profiles have hastened the mass introduction of gene technologies and, therefore, the defeat over many earlier incurablemore...

Mitochondria are the key players in cell metabolism, calcium homeostasis, and reactive oxygen species (ROS) production. Mitochondrial genome alterations are reported to be associated with numerous human disorders affecting nearly all tissues. In this review, we discuss the available information on the involvement of mitochondrial DNA (mtDNA) mutations in cell dysfunction.