Retroviral vectors encoding ADA regulatory locus control region provide enhanced T-cell-specific transgene expression.

ABSTRACT:

Background

Murine retroviral vectors have been used in several hundred gene therapy clinical trials, but have fallen out of favor for a number of reasons. One issue is that gene expression from viral or internal promoters is highly variable and essentially unregulated. Moreover, with retroviral vectors, gene expression is usually silenced over time. Mammalian genes, in contrast, are characterized by highly regulated, precise levels of expression in both a temporal and a cell-specific manner. To ascertain if recapitulation of endogenous adenosine deaminase (ADA) expression can be achieved in a vector construct we created a new series of Moloney murine leukemia virus (MuLV) based retroviral vector that carry human regulatory elements including combinations of the ADA promoter, the AD

SUBMITTER: Trinh AT 

PROVIDER: S-EPMC2809042 | biostudies-literature | 2009 Dec

REPOSITORIES: biostudies-literature