Technology ID

AAV5 Vector and Uses Thereof

Lead Inventor
Chiorini, John (Jay) (NHLBI)
Kotin, Robert (NHLBI)
Lead IC
The invention described and claimed in this patent application provides for novel vectors and viral particles which comprise adeno-associated virus serotype 5 (AAV5). AAV5 is a single-stranded DNA virus of either plus or minus polarity which, like other AAV serotypes (e.g., AAV4, AAV2) requires a helper virus for replication. AAV type 2 has the interesting and potentially useful ability to integrate into human chromosome 19 q 13.3-q ter. This activity is dependent on the non-structural, Rep, proteins of AAV2. The Rep proteins of AAV types 2 and 5 are dissimilar and are not able to substitute in DNA replication of the heterologous serotype. AAV5 offers several advantages which make it attractive for use in gene therapy: 1. increased production (10-50 fold greater than AAV2); 2. distinct integration locus when compared to AAV2; 3. Rep protein and ITR regions do not complement other AAV serotypes; and 4. appears to utilize different cell surface attachment molecules than those of AAV type 2.
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