Recombinant adeno-associated viral vector reference standards.

Abstract:

:Reference standard materials (RSMs) exist for a variety of biologics including vaccines but are not readily available for gene therapy vectors. To date, a recombinant adeno-associated virus serotype 2 RSM (rAAV2 RSM) has been produced and characterized and was made available to the scientific community in 2010. In addition, a rAAV8 RSM has been produced and will be characterized in the coming months. The use of these reference materials by members of the gene therapy field facilitates the calibration of individual laboratory vector-specific internal standards and the eventual comparison of preclinical and clinical data based on common dosage units. Normalization of data to determine therapeutic dose ranges of rAAV vectors for each particular tissue target and disease indication is important information that can enhance the safety and protection of patients.

journal_name

Methods Enzymol

journal_title

Methods in enzymology

authors

Moullier P,Snyder RO

doi

10.1016/B978-0-12-386509-0.00015-6

subject

Has Abstract

pub_date

2012-01-01 00:00:00

pages

297-311

eissn

0076-6879

issn

1557-7988

pii

B978-0-12-386509-0.00015-6

journal_volume

507

pub_type

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