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Application filed by Genzyme CorpfiledCriticalGenzyme Corp
Priority claimed from PCT/US2016/017210external-prioritypatent/WO2016130591A2/en
Publication of MA40818A1publicationCriticalpatent/MA40818A1/en
Publication of MA40818B2publicationCriticalpatent/MA40818B2/en
La présente invention concerne de nouveaux procédés d'administration de particules virales adéno-associées recombinées (raav) au système nerveux central d'un mammifère (p. Ex., l'homme). Selon certains aspects, les procédés impliquent l'administration des particules raav contenant un acide nucléique hétérologue au striatum et l'induction de l'expression de l'acide nucléique hétérologue dans au moins le cortex cérébral et le striatum du mammifère.The present invention provides novel methods of administering recombinant adeno-associated viral particles (raav) to the central nervous system of a mammal (e.g., humans). In some aspects, the methods involve administering raav particles containing striatal heterologous nucleic acid and inducing expression of the heterologous nucleic acid in at least the mammalian cerebral cortex and striatum.
MA40818A2015-02-102016-02-09
Improved delivery of viral particles to the striatum and cortex
MA40818B2
(en)