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Recombinant Adenoviral Vector System for Gene Therapy
Principal Investigator Robert Schneider, Ph.D., Department of Biochemistry New York University School of Medicine Gene therapy, the replacement or supplementation of defective genetic information by transfer of normal functional genes, has significant potential as a method for treating genetic disorders. Recent approaches for gene therapy have involved gene transfer using recombinant viral vectors, exploiting RNA and DNA tumor viruses - many of which are pathogenic and capable of causing disease. Retroviruses have also been extensively studied, however, these viruses are unstable, randomly integrate into the host genome (which can lead to insertional mutagenesis) and may activate protooncogene expression. Adeno-associated viruses, also being utilized, have the advantage of integrating in specific regions of the host genome. The usefulness of currently available retroviral and adeno-associated viral vector systems is limited by their inability to accept heterologous DNA fragments greater than 3 to 5 kilobases (kb) and to produce large quantities of viral stocks. Recombinant adenovirus vector systems can now only accommodate 6 to 7 kb of foreign DNA and, despite deletion of certain viral replication regions, do cause significant cytopathology at high multiplicities of infection. Description of the Project Dr. Schneider has developed a novel, trans-packaging adenovirus system which comprises (i) a recombinant adenovirus vector which contains cis-acting sequences required for replication and accommodates heterologous DNA and (ii) trans-acting viral functions necessary for replication and encapsidation of the recombinant vector into infectious adenovirus particles. The recombinant vector permits the insertion of large fragments of DNA (15 to 30 kb in size) while retaining the ability to be efficiently packaged. Unique aspects of this system reduce or eliminate the cytopathology usually observed with other adenoviral systems. Applications NYU is seeking an industrial partner to assist in further development and commercialization of this technology. This unique adenovirus vector system can be utilized for a variety of gene therapy applications, particularly where safe and efficient introduction of large DNA fragments is desired. Patent Status A patent application covering this novel adenovirus vector system has been filed in the United States. For further information please contact New York University Industrial Liaison/Technology Transfer 650 First Avenue, New York, N.Y. 10016 Tel: (212)263-8178 Fax: (212)263-8189 |
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