Use of lentiviral vectors expressing factor VIII

• Main Authors: CANTORE, ALESSIO, NALDINI, LUIGI, PETERS, ROBERT T, ANNONI, ANDREA, PATARROYO-WHITE, SUSANNAH, MOFFIT, JEFF, MILANI, MICHELA, LIU, TONGYAO, SEREGIN, ALEXEY, DRAGER, DOUGLAS
• Format: Patent
• Language: Chinese; English
• Published: 16.12.2019
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; SUGARS; VINEGAR; WINE

The present disclosure provides lentiviral vectors comprising codon optimized Factor VIII sequences, and methods of using such lentiviral vectors. The liver-targeted lentiviral vectors disclosed herein can be used for gene therapy, wherein the lentiviral gene delivery enables stable integration of t...