Modern gene therapy drugs

Abstract

Gene therapy is a modern and effective approach for treating diseases previously considered incurable by traditional methods. Its main strategy is to transfer the genetic material into the patient’s somatic cells to inhibit or promote the expression of a target gene/protein associated with disease development. This type of therapy is used in patients with few or no alternative treatment options.

Over the past two decades, gene therapy has produced promising clinical results, with many products approved for treating severe oncologic, hematologic, infectious, orphan, and/or inherited diseases, including monogenic and metabolic diseases. The scope of clinical indications and tissue targets for gene therapy is expanding every year.

This review examines new genetically engineered drugs recently introduced in global and domestic markets. These drugs are based on molecules of small interfering RNA (siRNA), mRNA, antisense nucleotides, and viral and plasmid vectors. Each group of drugs is unique and has its mechanism of action, depending on the purpose of the therapy.

Keywords

small interfering RNA / mRNA vaccines / antisense oligodeoxyribonucleotides / genetic vectors / plasmids

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[189] https://mioby.ru/ [Internet]. The “Circle of Goodness” purchased unregistered drugs for children with Duchenne muscular dystrophy [cited: 27.12.2022]. Available from: https://mioby.ru/novosti/krug-dobra-zakupil-nezaregistrirovannye-preparaty-dlja-detej-s-miodistrofiej-djushenna/ (In Russ).

[190] https://mioby.ru/ [интернет]. «Круг добра» закупил незарегистрированные препараты для детей с миодистрофией Дюшенна. Режим доступа: https://mioby.ru/novosti/krug-dobra-zakupil-nezaregistrirovannye-preparaty-dlja-detej-s-miodistrofiej-djushenna/ Дата обращения: 27.12.2022.

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[193] https://pharmvestnik.ru/ [Internet]. BIOCAD has submitted an application for clinical trials of the first Russian medicine for SMA [cited: 18.11.2022]. Available from: https://pharmvestnik.ru/content/news/BIOCAD-podala-zayavku-na-klinicheskie-ispytaniya-pervogo-rossiiskogo-lekarstva-ot-SMA.html (In Russ).

[194] https://pharmvestnik.ru/ [интернет]. BIOCAD подала заявку на клинические испытания первого российского лекарства от СМА. Режим доступа: https://pharmvestnik.ru/content/news/BIOCAD-podala-zayavku-na-klinicheskie-ispytaniya-pervogo-rossiiskogo-lekarstva-ot-SMA.html Дата обращения: 18.11.2022.

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