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1. WO2020223118 - MULTIPLEX SHRNA FOR USE IN VECTORS

Publication Number WO/2020/223118
Publication Date 05.11.2020
International Application No. PCT/US2020/029692
International Filing Date 24.04.2020
IPC
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants
  • DHARMACON, INC. [US]/[US]
Inventors
  • MACHADO, Hidevaldo Bueno
  • CHOU, Eldon Thomas
Agents
  • LOCKE, Scott
Priority Data
62/842,04902.05.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MULTIPLEX SHRNA FOR USE IN VECTORS
(FR) ARNSH MULTIPLEX DESTINÉ À ÊTRE UTILISÉ DANS DES VECTEURS
Abstract
(EN)
New polynucleotides and new expression vectors for multiplexed microRNA-based shRNAs are provided. The shRNAs generated from these polynucleotides and vectors can knock down the expression of multiple genes without undesirable levels of off-target effects.
(FR)
L'invention concerne de nouveaux polynucléotides et de nouveaux vecteurs d'expression pour des ARNsh à base de micro-ARN multiplexés. Les ARNsh générés à partir de ces polynucléotides et vecteurs peuvent inactiver l'expression de multiples gènes sans niveaux indésirables d'effets hors cible.
Latest bibliographic data on file with the International Bureau