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1. (WO2018183705) OXIDATION-RESISTANT AAT GENE THERAPY

Pub. No.:    WO/2018/183705    International Application No.:    PCT/US2018/025183
Publication Date: Fri Oct 05 01:59:59 CEST 2018 International Filing Date: Fri Mar 30 01:59:59 CEST 2018
IPC: A61K 31/712
A61K 48/00
C07K 14/81
C12N 15/00
C12N 15/09
C12N 15/63
Applicants: CORNELL UNIVERSITY
CRYSTAL, Ronald G.
STILES, Katie
SOSULSKI, Meredith
KAMINSKY, Stephen M.
SONDHI, Dolan
DE, Bishnu
ROSENBERG, Jonathan
Inventors: CRYSTAL, Ronald G.
STILES, Katie
SOSULSKI, Meredith
KAMINSKY, Stephen M.
SONDHI, Dolan
DE, Bishnu
ROSENBERG, Jonathan
Title: OXIDATION-RESISTANT AAT GENE THERAPY
Abstract:
A gene therapy treatment for alpha 1-antitrypsin (AAT) deficiency is provided comprising a plasmid or viral, e.g., an AAV, vector coding for an elastase- or cathepsin G-inhibiting, oxidation-resistant human AAT with a substitution at, for example, Met358 and/or Met351.