ISSN Print 2500–1094
ISSN Online 2542–1204
Bulletin of RSMU
BIOMEDICAL JOURNAL OF PIROGOV UNIVERSITY (MOSCOW, RUSSIA)
REVIEW
Recombinant adeno-associated viruses as a gene delivery vehicle for the use in molecular medicine
About authors
1 Kulakov National Medical Research Center for Obstetrics, Gynecology and Perinatology, Moscow, Russia
2 Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Pirogov Russian National Research Medical University, Moscow, Russia
Correspondence should be addressed: Natalia Usman
Oparina, 4, Moscow, 117997, Russia; ur.xednay@namsu.ailatan
About paper
Author contribution: NYu Usman — preparation of the manuscript; DV Rebrikov — editing of the manuscript.
Received: 2021-10-19
Accepted: 2021-10-29
Published online: 2021-10-31
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Viral mechanisms for the delivery of genetic material are widely used in molecular medicine. Recombinant adeno-associated viruses (rAAV) represent a promising tool for in vivo gene delivery. The review considers nosological spectrum, molecular mechanisms, the choice of drug administration route depending on target structures, the choice of serotype, and the methods of active ingredient manufacturing for rAAV-mediated gene therapy.
Keywords: gene therapy, adeno-associated viruses, AAV, rAAV