FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, in particular to a method of treating spinal muscular atrophy (SMA), comprising administering to a subject in need thereof, a therapeutically effective amount of recombinant virions of adeno-associated virus (rAAV), containing a self-complementary adeno-associated viral (scAAV) vector containing a heterologous nucleic acid construct.
EFFECT: invention provides effective treatment of spinal muscular atrophy (SMA).
6 cl, 10 dwg, 4 ex
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