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.
Department of Cell Biology, Erasmus MC, Erasmus University, P.O. Box 1738, Rotterdam DR 3000, The Netherlands.
Daniel
D
Klink
Dirk
D
Schindelhauer
Andreas
A
Laner
Torry
T
Tucker
Zsuzsanna
Z
Bebok
Erik M
EM
Schwiebert
A Christopher
AC
Boyd
Bob J
BJ
Scholte
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