COMPOSITION AND METHODS FOR HIGHLY EFFICIENT GENE TRANSFER USING AAV CAPSID VARIANTS Russian patent published in 2019 - IPC C12N15/864 C12N7/01 C07K14/15 A61K48/00 

Abstract RU 2683497 C2

FIELD: biotechnology.

SUBSTANCE: described is an adeno-associated virus (AAV) vector for delivering a heterologous nucleic acid sequence to a subject, comprising a VP1 capsid protein, which contains one or more lysine substitutions, where one lysine replacement is K137R. Said vector additionally contains a minigene comprising AAV inverted terminal repeats and a heterologous nucleic acid sequence operably linked to regulatory sequences which direct expression of a product from the heterologous nucleic acid sequence in a host cell, said lysine substitution being effective to inhibit ubiquitination of said capsid protein. Application of the claimed vector is also described.

EFFECT: transduction of said AAV vector in the target cell increases.

29 cl, 9 tbl, 1 ex, 20 dwg

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RU 2 683 497 C2

Authors

Yazisioglu Mustafa N.

Mingozzi Federiko

Angela Khaver

Khaj Ketrin A.

Dates

2019-03-28Published

2013-04-18Filed