FIELD: biotechnology.
SUBSTANCE: described is a group of inventions involving a method for increasing the level of expression of a transgene in tissue and a recombinant viral vector for use in treating methylmalonic acidemia (MMA). In one embodiment, the method involves a step of administering to an individual in need thereof a composition which delivers a transgene which is integrated into the genome of at least a cell population in the individual’s tissue.
EFFECT: invention extends the range of agents for enhancing the expression of the transgene in tissue.
18 cl, 32 dwg, 4 tbl, 18 ex
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|
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|
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