New reference material to standardize gene therapy protocols in clinical trials

Mary Ann Liebert, Inc./Genetic Engineering NewsOct 7 2010

The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com). The article, which is published online ahead of print, is available free online at www.liebertpub.com/hum

The growing popularity in the gene therapy community of using recombinant adeno-associated virus (rAAV) vectors as vehicles to transfer a gene of interest into a host cell has led to increasing numbers of human clinical trials and animal studies with rAAV. In the past, the absence of standardization-such as for rAAV potency and dosing-have made it difficult for researchers and clinicians to compare their protocols and the results of gene therapy experiments. The availability of this rAAV reference standard will allow vector parameters to be expressed in common units, vector doses administered by different investigators to be normalized to an agreed-upon standard, and laboratories to calibrate their internal standards to a common reference material.

Martin Lock, from the Gene Therapy Program at the University of Pennsylvania (Philadelphia) and colleagues from several major medical and research institutions around the world, describe the development of reference standard in the article "Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material."

The American Type Culture Collection will make the rAAV2 reference standard material available to the scientific community. AAV serotype 2 was selected as the basis for the reference material because it is the best characterized AAV subtype. The material has been thoroughly analyzed and found to be free of microbial contamination. During development of the rAAV2 reference standard samples were distributed to 16 laboratories worldwide for extensive analysis and characterization.

"The development of a reference standard for an AAV vector is an important step towards the eventual commercial development of this important vector platform," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, in Philadelphia.