Gene and cell therapy for cystic fibrosis: From bench to bedside
| Autor: | Massimo Conese, A. Christopher Boyd, Joseph Rosenecker, Stefaan C. De Smedt, Bob J. Scholte, Fiorentina Ascenzioni, Joanna Rejman, Charles Coutelle, Dirk Schindelhauer, Ida De Fino |
|---|---|
| Přispěvatelé: | Cell biology |
| Rok vydání: | 2011 |
| Předmět: |
CFTR GENE
Pulmonary and Respiratory Medicine Human artificial chromosome Genetic enhancement Genetic Vectors Stem cells Vectors in gene therapy Gene delivery PLACEBO-CONTROLLED TRIAL Cystic fibrosis Chromosomes Artificial Human In utero gene therapy MESENCHYMAL STEM-CELLS Cell therapy SDG 3 - Good Health and Well-being Animals Humans Medicine Animal model Pediatrics Perinatology and Child Health ARTIFICIAL CHROMOSOME FORMATION CFTR cystic fibrosis stem cells gene delivery animal model vector development human artificial chromosome in utero gene therapy cftr Gene IN-VIVO Vector development biology business.industry Genetic Therapy medicine.disease BONE-MARROW-TRANSPLANTATION DEPENDENT SPATIAL ARRANGEMENTS AIRWAY EPITHELIAL-CELLS Cystic fibrosis transmembrane conductance regulator Disease Models Animal TRANSMEMBRANE CONDUCTANCE REGULATOR MONKEYS MACACA-MULATTA Pediatrics Perinatology and Child Health Immunology biology.protein Cancer research Stem cell business Stem Cell Transplantation |
| Zdroj: | Journal of Cystic Fibrosis, 10, S114-S128. Elsevier Conese, M, Ascenzioni, F, Boyd, A C, Coutelle, C, De Fino, I, de Smedt, S, Rejman, J, Rosenecker, J, Schindelhauer, D & Scholte, B J 2011, ' Gene and cell therapy for cystic fibrosis: From bench to bedside ', Journal of Cystic Fibrosis, vol. 10, pp. S114-S128 . https://doi.org/10.1016/S1569-1993(11)60017-9 |
| ISSN: | 1569-1993 |
| DOI: | 10.1016/s1569-1993(11)60017-9 |
| Popis: | Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithelial cells. However, the limited efficacy of gene transfer vectors as well as extra- and intracellular barriers have prevented the development of a gene therapy-based treatment for CF. Here, we review the use of new viral and nonviral gene therapy vectors, as well as human artificial chromosomes, to overcome barriers to successful CFTR expression. Pre-clinical studies will surely benefit from novel animal models, such as CF pigs and ferrets. Prenatal gene therapy is a potential alternative to gene transfer to fully developed lungs. However, unresolved issues, including the possibility of adverse effects on pre- and postnatal development, the risk of initiating oncogenic or degenerative processes and germ line transmission require further investigation. Finally, we discuss the therapeutic potential of stem cells for CF lung disease. (C) 2011 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved. |
| Databáze: | OpenAIRE |
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