Work

Prospects of gene therapy with hematopoietic stem cells

Year: 2024

Type: article

Abstract: Gene therapy is subdivided into in vivo and ex vivo according to the tactics of delivery of therapeutic constructions. In vivo therapy, a vector containing a therapeutic construction is injected into the patient. In ex vivo therapy, cells are removed from the patient's body, genetically modified, and then returned. In ex vivo therapy for inherited diseases, hematopoietic stem cells are most often subjected to modification. Despite the advantage of in vivo therapy, which consists in the possibility of scale-up of production and convenience of use of ready-to-use drug, therapy based on transplantation of genetically modified hematopoietic stem cells remains relevant for a number of genetic diseases.

Source: Bulletin of Russian State Medical University

DOI: 10.24075/brsmu.2024.074

Authors: YM Valieva, KV Popov

Institutions: Moscow Institute of Physics and Technology, National Medical Research Center for Obstetrics, Gynecology and Perinatology named after Academician V.I.Kulakov of the Ministry of Healthcare of the Russian Federation

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: diamond

Funder: Ministry of Health of the Russian Federation