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1. (WO2018089527) FRATAXIN EXPRESSION CONSTRUCTS

Pub. No.:    WO/2018/089527    International Application No.:    PCT/US2017/060680
Publication Date: Fri May 18 01:59:59 CEST 2018 International Filing Date: Thu Nov 09 00:59:59 CET 2017
IPC: C12N 15/86
A61K 48/00
C07K 14/47
Applicants: INTREXON CORPORATION
Inventors: SCHAUER, Stephen
THOMAS, Darby
ROBINSON, Gregory
PYKETT, Mark
THORN, Richard
GRUIS, Kirsten
Title: FRATAXIN EXPRESSION CONSTRUCTS
Abstract:
The invention provides polynucleotides, vectors and viruses expressing frataxin and methods of treating Friedreich's Ataxia.