Genetically Engineered AAV Rep for Gene Targeting and rAAV Production

A genetic element within the Rep gene sequence, which is inhibitory to Ad replication. Background: Gene therapy strategies have exploited lenti or retro –viral approaches for long-term gene replacement, however, their clinical applications remain limited because of potential for viral-associated oncogenesis. Recently, gene therapy strategies have attempted to use hybrid Adenovirus/Adeno-associated viruses (Ad/AAV) to combine the capacity, tropism and ease of production of adenovirus (Ad) with adeno-associated virus’s (AAV’s) ability for site-specific integration (SSI). Although the AAV Rep78 protein is required for SSI, it also has an inhibitory effect on Ad replication, particularly when co-expressed within the Ad backbone leading difficulty in generating and integrating transgene within the back-bone of a single hybrid virus. Technology Overview: Researchers at Stony Brook University have identified a genetic element within the Rep gene sequence, which is inhibitory to Ad replication. This AAV Rep gene was then genetically recoded using synonymous codon pair re-engineering to overcome Rep’s inhibitory effects on Ad replication. The novel construct thus created provides a unique opportunity to place all genetic elements in one virus for the purpose of safely integrating a transgene into a specific region of the human genome. Advantages: Combines the advantages of Ad (high titer, high infectivity and large capacity) and integration capability of AAV

Offers one-step packaging systems for rAAV viral production. Applications: New Gene therapy vectors

Improved rAAV production methods. Intellectual Property Summary: Patent application submitted Stage of Development: Pending US Utility covering compositions and methods of use Licensing Potential: Licensing,Commercial partner,Development partner,Seeking investment Licensing Status: Available for License. Stony Brook University seek to develop and commercialize, by an exclusive or non-exclusive license agreement and/or sponsored research, with a company active in the area. R-8286 Additional Information: therapies,virus,viral,adenovirus,adeno-associated virus,proteins,genetic,genome,high titer,gene therapy vector,gene therapy,gene therapeutic,gene therapy development,lentiviral gene therapy,adeno associated viral vector,aav gene therapy,site specific integration,oncogenesis,hybrid virus,transgene Please note, header image is purely illustrative. Source: rost9, stock.adobe.

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