Gene delivery systems--gene therapy vectors for cystic fibrosis.
Gene delivery systems (GDS) play a central role in the development of gene therapy strategies for Cystic Fibrosis (CF). Further, these systems are important tools in studies with cultured cells and in animal models. In this review, we describe the properties of several viral and synthetic gene delivery systems, and evaluate their possible application in gene therapy of CF. While many gene delivery systems give satisfactory results in cultured or animal studies, none of these systems has been shown to fulfil all the requirements of safety and efficacy for use in CF patients. The intact airway epithelium, the most important target in CF gene therapy, proves to be well protected against invading vector systems.
Klink
Daniel
D
Department of Cell Biology, Erasmus MC, Erasmus University, P.O. Box 1738, Rotterdam DR 3000, The Netherlands.
Schindelhauer
Dirk
D
Laner
Andreas
A
Tucker
Torry
T
Bebok
Zsuzsanna
Z
Schwiebert
Erik M
EM
Boyd
A Christopher
AC
Scholte
Bob J
BJ
D000256:Adenoviridae; D000818:Animals; D002450:Cell Communication / Q000502:physiology; D003550:Cystic Fibrosis / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D000229:Dependovirus; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D016086:Lentivirus; D023421:Models, Animal; D020545:Respiratory Mucosa / Q000502:physiology*; D029082:Sendai virus; D014162:Transfection
C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator