Targeted Gene Modification by Parvoviral Vectors

Introduction Though the development of integrating vectors based on eukaryotic viruses has made possible the efficient introduction of genes into mammalian chromosomes, there are many situations where it would be preferable to modify specific chromosomal sequences. This ability could eliminate unwanted chromosomal genotypes and avoid position effects on gene expression. The need for such an ability to modify a preexisting locus is particularly acute in gene therapy, where mutant genes can have dominant effects and tissue-specific controls on expression are often critical. Thus, a need exists for methods of obtaining specific genetic modification at selected target sites in vertebrate cellular genomes at high frequencies. Technology description Researchers at the University of Washington have developed a method of effecting gene therapy via homologous recombination. A recombinant parvoviral vector is used to modify a gene in a vertebrate cell at a pre-selected target locus. This method makes possible precise modifications of the genome of a cell, avoiding undesired effects such as disruption of a desirable gene by insertion of an exogenous gene. The modification can include one or more deletions, insertions, substitutions, or a combination thereof. Business Opportunity More than thirty different acquired, monogenic, multigenic, short-term, and chronic diseases, including cancer, cardiovascular disease, hemophilia A & B, cystic fibrosis, graft-vs.-host, and diabetes, have gene therapy treatments being researched and developed where the total potential dollar value of the gene therapy markets are billions of dollars. Intellectual Property Position

Type of Offer: Licensing



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