FIELD: biotechnologies.
SUBSTANCE: disclosed are the disease treatment biotechnological methods or the therapeutic agent delivering to the mammal. Into the mammal cerebellum-cerebral cistern and / or ventricle introducing the rAAV particles containing vector comprising therapeutic protein encoding nucleic acid inserted between the pair of AAV inverted terminal repeats, so, that having access to cerebrospinal fluid (CSF) cells are expressing the therapeutic agent and in some embodiments secrete the therapeutic agent into CSF for propagation in the brain.
EFFECT: invention extends the drug delivery to the nervous system means range.
22 cl, 17 dwg, 2 ex
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