A.G. Kirillov¹, E.A. Guryanova^{1, 2}, A.V. Golenkov^{1, 2}

 ¹Institute for Advanced Training of Physicians, Cheboksary

²Chuvash State University named after I.N. Ulyanov, Cheboksary

Contact details:

Kirillov A.G. — MD, Chief Researcher

Address: 27 Sespelya St., 428018 Cheboksary, Russian Federation, tel.: +7 (8352) 70-92-42 (ext. 224), e-mail: giduv-muz@med.cap.ru

The review is devoted to modern developments in the treatment of osteopetrosis, the autosomal-recessive form of which has a high endemicity for the indigenous population of Chuvash and Mari El Republicы. Autosomal-recessive osteopetrosis (ARO) is a major medical and social problem, primarily due to the severity of clinical manifestations, low efficiency of pathogenetic therapy and the limitations of etiotropic therapy methods. Until now, the generally accepted standard for treating the autosomal-recessive form is transplantation of pluripotent hematopoietic stem cells. However, information about the development of alternative ARO treatment methods based on innovative technologies appears in medical publications. Therefore, the study of alternative methods of treating this disease is relevant.

Key words: autosomal-recessive osteopetrosis, hematopoietic stem cell transplantation, alternative treatment methods, gene therapy, lentiviral correction.

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