Adenoviruses have been characterized extensively since their initial description in the early
1950s (Hillemann & Werner, 1954; Rowe et al., 1953) and there is now a panoply of …

Last-generation adenovirus vectors, also called helper-dependent or gutless adenovirus,
are very attractive for gene therapy because the associated in vivo immune response is …

Gene replacement therapy is an attractive approach for treatment of genetic disease, but
may be complicated by the risk of a neutralizing immune response to the therapeutic gene …

Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation
factor IX (FIX). Using adeno-associated virus (AAV)–mediated, liver-directed gene therapy …

The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the
past decade, with the advent of novel capsid serotype and organ-specific promoters, and an …

We systematically compared human factor IX gene expression from a variety of plasmids
containing different cis-regulatory sequences after transfection into different hepatocyte cell …

Recombinant adeno-associated virus (rAAV) vectors have emerged as highly promising for
use in gene transfer for a variety of reasons, including lack of pathogenicity and wide host …

High-titer self-inactivating human immunodeficiency virus type-1 (HIV-1)–based vectors
expressing the green fluorescent protein reporter gene that contained the central polypurine …

Viral vector is the most effective means of gene transfer to modify specific cell type or tissue
and can be manipulated to express therapeutic genes. Several virus types are currently …

It has become apparent that the clinical success anticipated in the field of gene therapy has
been limited by progress in several of the fundamental areas of genetics, molecular and …