Treatment of Genetic Diseases: Current Trends in the Development of Biomedical Cell Products

Abstract

Genetic diseases are often progressive in nature, and without proper treatment may result in disability or death. Difficulties with diagnosis of genetic diseases and lack of effective treatment are global public health challenges. Medical care for patients with genetic diseases is often confined to symptomatic and palliative care. Starting from the 2000s, great hopes have been placed on cell-based medicinal products (which are referred to as biomedical cell products in the Russian legislation) and gene therapy products. The aim of the study was to review current trends in the development of biomedical cell products for the treatment of genetic diseases. The paper focuses on cell-based products for the treatment of monogenic genetic diseases, such as severe combined immunodeficiency (SCID), recessive dystrophic epidermolysis bullosa (RDEB), beta-haemoglobinopathies, alpha-1-antitrypsin deficiency, haemophilia A, and Duchenne muscular dystrophy. Such drugs are being developed in many countries and are now entering preclinical and different stages of clinical trials. Products based on various types of viable cells, including differentiated cells, stem cells, induced pluripotent cells, as well as cells genetically modified ex vivo, may be developed for the treatment of one and the same disease. The main priority is the creation of such products that will obviate the need for replacement therapy or palliative care, and that will significantly increase life expectancy and quality of life.