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1. (WO2018200580) SELECTABLE STEM CELL MODIFICATION FOR GENE THERAPY USING TRANSIENT CELL SURFACE MARKING OF MODIFIED CELLS USING MODIFIED CD4 MOLECULE

Pub. No.:    WO/2018/200580    International Application No.:    PCT/US2018/029213
Publication Date: Fri Nov 02 00:59:59 CET 2018 International Filing Date: Wed Apr 25 01:59:59 CEST 2018
IPC: C07K 14/73
C07K 16/28
G01N 33/543
G01N 33/563
C12N 15/867
C12N 15/86
C12N 5/071
A61K 48/00
Applicants: TEXAS TECH UNIVERSITY SYSTEM
GARG, Himanshu
JOSHI, Anjali
Inventors: GARG, Himanshu
JOSHI, Anjali
Title: SELECTABLE STEM CELL MODIFICATION FOR GENE THERAPY USING TRANSIENT CELL SURFACE MARKING OF MODIFIED CELLS USING MODIFIED CD4 MOLECULE
Abstract:
Aa gene therapy method comprises modifying at least one CD4 molecule to form CD4mod, marking target cells with immunomagnetic beads, and killing the marked target cells. The method comprises applying a first vector that expressesTK-SR39 and applying a second vector that expresses HIV Tat and a CRISPR-CCR5 cassette to knockout CCR5. TheTK- SR39 rapidly kills cells in a presence of Ganciclovir.