Modified IX factor and composition, method and use for gene transfer to cell, organ and tissue

摘要

A vector encoding a modified IX factor for the IX factor nucleic acid for cells, tissues or organs for expression (transcription) of IX factors, vectors such as expression cassettes, viruses (e.g., lentivirus or adeno associated virus) vectors, etc And method for introducing gene.Nucleic acid sequence encoding human IX factor protein, nucleic acid is a nucleic acid sequence with a number of CpG dinucleotides reduced compared to the wild type sequence encoding human IX factor.No selection