Research Centre for Medical Genetics
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Moscow 115522, Russian Federation
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The laboratory of Genome Editing of the Research Centre for Medical Genetics proposed an algorithm for selecting methods for the purification of adeno-associated viral vectors for gene therapy drugs

The algorithm was developed based on the analysis of data from almost 100 scientific publications devoted to all purification methods available today. An article on the algorithm was published in the “BioEssays” journal (Q1).

Recombinant adeno-associated viruses (rAAV) are promising vectors for the delivery of various genetic constructs to eukaryotic cells.

rAAV have properties that allow them to be used both in vitro and in vivo: they are nearly non-pathogenic, safe, do not cause an immune response and other side reactions. rAAV can accurately deliver the necessary material to target cells.

Purification and concentration of rAAV vectors play a critical role in achieving high viral titer, stability, efficacy and purity of future preparations.

- Existing cleaning methods differ markedly from each other in terms of mechanisms, efficiency, laboriousness and cost. Today researchers choose the right type of methodology mainly by trial and error. The algorithm that we propose allows to simplify this choice and stop at the best method suitable for the purposes of the experiment, while taking into account the resources of the laboratory. For example, if it is planned to manufacture GMP for preclinical testing of the drug on animals, then we should come up with purification methods that will allow obtaining a highly purified drug. An example of such a method is the chromatography technique, - explained Svetlana Smirnikhina, Head of the Laboratory of Genome Editing, Ph.D.

Recombinant adeno-associated viruses are used as delivery methods for innovative drugs such as Zolgensma (a drug for the treatment of spinal muscular atrophy) and Luxturna (a drug for the treatment of hereditary retinal dystrophy caused by a mutation in the RPE65 gene).