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1. (WO2018204694) IMPROVED LENTIVIRUSES FOR TRANSDUCTION OF HEMATOPOIETIC STEM CELLS.

Pub. No.:    WO/2018/204694    International Application No.:    PCT/US2018/030956
Publication Date: Fri Nov 09 00:59:59 CET 2018 International Filing Date: Fri May 04 01:59:59 CEST 2018
IPC: C12N 7/00
Applicants: BIOMARIN PHARMACEUTICAL INC.
Inventors: LOCHRIE, Michael
YONEMOTO, Wes
ANKALA, Ramya
LUNA, Jac, Michael
Title: IMPROVED LENTIVIRUSES FOR TRANSDUCTION OF HEMATOPOIETIC STEM CELLS.
Abstract:
Recombinant viruses, comprising a lentiviral vector carrying a heterologous transgene, packaged in an envelope containing at least one heterologous envelope protein, are described. Also described are methods of producing these recombinant viruses and methods of using these viruses to deliver genes to selected target cells. These recombinant viruses are particularly useful for transducing a hematopoietic stem cells, in particular CD34+ cells.