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1. (WO2018007871) MATERIALS AND METHODS FOR TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS

Pub. No.:    WO/2018/007871    International Application No.:    PCT/IB2017/000987
Publication Date: Fri Jan 12 00:59:59 CET 2018 International Filing Date: Sat Jul 08 01:59:59 CEST 2017
IPC: C12N 15/113
C12N 15/63
A61P 43/00
Applicants: CRISPR THERAPEUTICS AG
Inventors: BOGORAD, Roman, Lvovitch
STEBBINS, Jeffrey, Williams
GREGOIRE, Francine, Marie
COWAN, Chad, Albert
LUNDBERG, Ante, Sven
PADMANABHAN, Hari
Title: MATERIALS AND METHODS FOR TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS
Abstract:
Materials and methods for treating a patient with Transthyretin Amyloidosis, both ex vivo and in vivo, are provided, and materials and methods for modulating the expression, function, or activity of a Transthyretin (TTR) gene in a cell by genome editing are provided.