PROPAGATION OF RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR

Award Information
Agency: Department of Health and Human Services
Branch: N/A
Contract: N/A
Agency Tracking Number: 19024
Amount: $500,000.00
Phase: Phase II
Program: SBIR
Awards Year: 1993
Solitcitation Year: N/A
Solitcitation Topic Code: N/A
Solitcitation Number: N/A
Small Business Information
Applied Immunesciences Inc.
200 Constitution Drive, Menlo Park, CA, 94025
Duns: N/A
Hubzone Owned: N
Woman Owned: N
Socially and Economically Disadvantaged: N
Principal Investigator
 Jane S Lebkowski
 (415) 326-7302
Business Contact
Phone: () -
Research Institution
N/A
Abstract
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.

* information listed above is at the time of submission.

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