Article
Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2008
Buckley Suzanne M K, Waddington S N, Jezzard S, Bergau A, Themis M, MacVinish L J, Cuthbert A W, Colledge W H, Coutelle C
Abstract excerpt
Due to its early onset and severe prognosis, cystic fibrosis (CF) has been suggested as a candidate disease for in utero gene therapy. In 1997, a study was published claiming that to how transient prenatal expression of CF transmembrane conductance regulator (CFTR) from an in utero-injected adenovirus vector could achieve permanent reversal of the CF intestinal pathology in adult CF knockout mice, despite the...
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