PROPAGATION OF RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR

RECOMBINANT ADENO-ASSOCIATED VIRUS (RAAV) VECTORS HAVE BEEN USED TO INTRODUCE GENETIC MATERIAL INTO A VARIETY OF CELL TYPES AND ARE A GOOD CANDIDATE FOR MULTIPURPOSE, SAFE DNA TRANSDUCTION SYSTEM. THE SPACIFIC AIM OF THE RESEARCH IS TO DEVELOP A SYSTEM TO VERY SIMPLY PRODUCE HIGH TITER RECOMBINANT AAV STOCKS. TO DATE, PRODUCTION OF RECOMBINANT AAV VECTORS IS A CUMBERSOME PROCESS, REQUIRING INEFFICIENT TRANSFECTIONS AND REPEATED MANIPULATIONS EACH TIME RECOMBINANT VIRUS STOCKS ARE DESIRED. THE WORK WILL TAKE ADVANTAGE OF CERTAIN MAMMALIAN GENE EXPRESSION SYSTEMS AND EPISOMAL MAINTENANCE VECTORS TO ESTABLISH GENERIC SYSTEMS TO EASILY PRODUCE ANY RAAV VECTOR IN LARGE QUANTITY. IF THIS RESEARCH IS SUCCESSFUL, THE LONG-TERM GOAL OF THE PROJECT IS TO INCORPORATE INTO THESE VECTORS, NOVEL EXPRESSION SYSTEMS IN ORDER TO REGULATE ACTIVITY OF THE INTRODUCED GENES IN SPECIFIC TARGETED CELL TYPES. THE COMMERCIAL POTENTIAL OF THESE VIRUSES AND THEIR PROPAGATION SYSTEM IS LARGE AND RANGES FROM THE USE OF THESE VECTORS TO MODIFY CELLS IN VITRO FOR THE PRODUCTION OF ANY PROTEIN PRODUCT TO THE GENETIC MODIFICATION OF A PATIENT'S CELLS AS A THERAPEUTIC TREATMENT.