A novel hybrid virus for gene therapy for long term expression of large DNA segments

A novel hybrid virus for gene therapy for long term expression of large DNA segments

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.

DESCRIPTION 
Dr. Wadie Bahou and colleagues 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 l Offers one-step packaging systems for rAAV viral production

APPLICATION 

New Gene therapy vectors l Improved rAAV production methods

DEVELOPMENT STAGE 
Proof of concept data is available.
PATENT STATUS 

Pending US Utility covering compositions and methods of use.

ADDITIONAL INFORMATION

INVENTORS 

Wadie Bahou M.D.
Professor of Medicine

Patrick Hearing Ph. D.
Professor

Varsha Sitaraman
Graduate Student

CASE MANAGER 

Sean Boykevisch, Ph. D.
sean.boykevisch@stonybrook.edu
6316326952

LICENSING STATUS 

Available for License

LICENSING POTENTIAL 
We seek to develop and commercialize, by an exclusive or non-exclusive license agreement and/or sponsored research, with a company active in the area.
TECHNOLOGY ID 
8286
PRIMARY KEY 
288830
KEYWORDS 

Adenovirus l Adeno-associated viruses l Rep78 Protein

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