FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, specifically to gene therapeutic preparations based on SMN1 gene, and can be used in medicine in therapy of spinal muscular atrophy (SMA). Disclosed is an isolated nucleic acid for producing a gene therapeutic viral preparation for SMN1 gene expression in mammalian target cells, which contains in direction from 5'-end to 3'-terminal nucleic acid coding SMN1 protein, and nucleic acid coding microRNA miR-23a. Invention also discloses an expression cassette and a vector containing said nucleic acid, as well as a recombinant virus based on AAV9 (adeno-associated virus of serotype 9).
EFFECT: invention provides enhanced functional action of SMN1 in vitro when using SMN1 gene in combination with miR-23a as a part of a gene therapeutic agent, which can be used in treating SMA.
12 cl, 5 dwg, 5 tbl, 4 ex
| Title | Year | Author | Number |
|---|---|---|---|
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|
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|
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|
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|
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|
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|
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|
RU2811445C2 |
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|
RU2751592C2 |
