Utility of the nasal model in gene transfer studies in cystic fibrosis.
Despite advances in the treatment for cystic fibrosis (CF), life expectancy for affected patients remains dramatically curtailed. Recent years have produced a spectacular increase in our understanding of the genetic, molecular and physiological bases of this disease. Gene transfer is a new and conceptually-attractive potential treatment for CF. A number of centres have undertaken preliminary human gene-therapy trials. Central to these trials has been the use of the nasal model in gene transfer studies. While the eventual target of gene therapy in CF will be the lungs, the nasal administration of vector offers a number of advantages over the tracheobronchial tree in early experimentation. Implicit in the use of the nasal model is the potential for rhinologic variables to influence the results. We review our own gene transfer studies as well as series from other institutions, considering the role of nasal factors in the experiments' outcomes. Rhinologic variables may, at least partially, potentially explain the sometimes disparate results reported in this emerging area of scientific interest.
Graham
S M
SM
Department of Otolaryngology/Head and Neck Surgery, University of Iowa College of Medicine, Iowa City, USA.
Launspach
J L
JL
D000256:Adenoviridae / Q000235:genetics; D000281:Administration, Intranasal; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors; D006801:Humans
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator