Research Centre for Medical Genetics
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Moscow 115522, Russian Federation
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An illustration from an article by RCMG researchers is on the cover of a highly rated journal

The Genome Editing Laboratory researchers have published a comprehensive review on modification of adenoassociated viral vectors for the treatment of lung diseases. Gene therapy for genetically determined diseases, including some pathology conditions of the respiratory system, requires an effective method of transgene delivery. Recombinant adenoassociated viral (rAAV) vectors are well studied and used in gene therapy because they are relatively simple, have low immunogenicity and can efficiently transduce eukaryotic cells. Today, many natural and artificial (with modified capsids) serotypes of AAV have been isolated, demonstrating preferential tropism to various tissues and cells according to the predominant receptors on the cell surface. The review describes various approaches to targeting rAAV to epithelial cells of the respiratory system, for example, using different types of capsid modifications and regulation of transgene expression by tissue-specific promoters. Part of the review is devoted to the transduction of lung stem and progenitor cells with AAV which is currently a challenging task.

Kochergin-Nikitsky K, Belova L, Lavrov A, Smirnikhina S. Tissue and cell-type-specific transduction using rAAV vectors in lung diseases. J Mol Med (Berl). 2021 Aug;99(8):1057-1071. doi: 10.1007/s00109-021-02086-y.