Site-specific integration with phiC31 integrase for prolonged expression of therapeutic genes.

摘要: Need of a site-specific integrating vector in gene therapy has become pressing, as recent work shown that many the current vectors used preferentially integrate vicinity genes. A would reduce risk insertional mutagenesis posed by randomly vectors, and non-viral safety immunogenicity problems associated with viral vectors. The phiC31 integrase is protein from Streptomyces phage been developed vector. catalyzes integration plasmid containing attB into pseudo attP sites mammalian genomes. It to function tissue culture cells well mice. Vectors based on were able treat tyrosinemia type I mouse model two forms epidermolysis bullosa keratinocytes patients, demonstrating its effectiveness Development integrase-based still underway, but it already provide long-term expression through integration.