Viral Vector Reference Material Available for Standardization of Gene Therapy Studies

An international consortium of research laboratories has characterized a reference adeno-associated virus (rAAV2) vector to be used for calibrating and standardizing experiments and clinical studies.

The reference material was produced and purified by a group headed by the University of Pennsylvania (Philadelphia, USA) employing helper virus–free transient transfection and chromatographic purification. The purified bulk material was distributed into vials, confirmed negative for microbial contamination, and then distributed for characterization along with standard assay protocols and assay reagents to 16 laboratories worldwide.

Details of the protocols and results of the characterization studies were published in the October 2010 online edition of the journal Human Gene Therapy. Using statistical transformation and modeling of the raw data, mean titers and confidence intervals were determined for capsid particles, vector genomes, transducing units, and infectious units. Further analysis confirmed the identity of the reference material as AAV2 and the purity relative to nonvector proteins as greater than 94%.

The rAAV2 reference standard has been deposited with the American Type Culture Collection (Manassas, VA, USA) and is available to the scientific community to calibrate laboratory-specific internal titer standards.

Related Links:
University of Pennsylvania
American Type Culture Collection