Position-Specific Insertion Vectors and Method of Using Same

Background: Retrovirus vectors infect cells at high efficiency and are capable of integrating DNA copies of themselves into a host genome. Such vectors are, in many ways, desirable for introduction and expression of exogenous DNA sequences in animal cells. In gene therapy or in the production of transgenic animals, prolonged, stable expression of exogenously introduced genes is a requirement in order for the treatment to become effective. This requirement stems from certain pitfalls or inefficiencies with present technology and, perhaps as a consequence, such prolonged treatment carries other certain distinct problems as well.

A few of the problems associated with the current state of the art are: 1. Some of the retroviral vectors insert in a region of the host genome which will suppress expression of the genes carried by the vector. 2. Some retroviral vectors could actually disrupt such expression and in so doing are mutagenic. 3. Insertion of some vectors may activate cellular oncogenes. 4. Some vectors may recombine with endogenous viruses and generate replication-competent retroviruses which would have the ability to multiply in the host cell in which they are directed to integrate. Technology: By introducing certain sequences into the retrovirus, researchers at the University of California, Irvine, have developed a vector with position specific insertion capability. Because this vector would insert into a specific innocuous location of the genome, disruption of host cell functions would be avoided. This breakthrough would address problems caused by random insertion. There would also be no disruption of expression and resultant mutagenesis. There would also be no risk of oncogene expression and the overall predictability of gene expression would be substantially improved. Application: This invention, therefore, once properly commercialized, has the potential to significantly advance the state of gene therapy or related applications

US 5,482,853

Type of Offer: Licensing

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