FIELD: biotechnology.
SUBSTANCE: group of inventions relates to methods for measurement of quantitative signs of gene-therapeutic vector preparations. A method for quantitative determination of a dose of AAV preparation is disclosed, providing quantitative determination of the total number of AAV capsids in AAV preparation or AAV fraction used for obtainment of AAV preparation, using AAV-specific ELISA analysis, and assessment of percentage or ratio of full and empty (full:empty) AAV capsids in AAV preparation or AAV fraction, using cryogenic translucent electronic microscopy (hereinafter – CryoTEM). A method for measurement of a concentration of hollow AAV capsids in AAV preparation, methods for administration of AAV preparation to a subject who needs it, and a method for the production of AAV preparation are also disclosed, each of which includes stages of AAV-specific ELISA analysis and CryoTEM.
EFFECT: group of inventions provides highly reliable and more accurate determination of a concentration, dose, and/or activity of AAV preparation.
44 cl, 16 dwg, 9 tbl, 4 ex
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