发明公开
EP2691529A1 METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES 审中-公开
治疗脑部疾病的方法和组合物

METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
摘要:
The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease.
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