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Testing gene therapy vectors in human primary nasal epithelial cultures

التفاصيل البيبلوغرافية
العنوان: Testing gene therapy vectors in human primary nasal epithelial cultures
المؤلفون: Huibi Cao, Hong Ouyang, Wan Ip, Kai Du, Wenming Duan, Julie Avolio, Jing Wu, Cathleen Duan, Herman Yeger, Christine E Bear, Tanja Gonska, Jim Hu, Theo J Moraes
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)
بيانات النشر: Elsevier, 2015.
سنة النشر: 2015
المجموعة: LCC:Genetics
LCC:Cytology
مصطلحات موضوعية: Genetics, QH426-470, Cytology, QH573-671
الوصف: Cystic fibrosis (CF) results from mutations in the CF transmembrane conductance regulator (CFTR) gene, which codes for a chloride/bicarbonate channel in the apical epithelial membranes. CFTR dysfunction results in a multisystem disease including the development of life limiting lung disease. The possibility of a cure for CF by replacing defective CFTR has led to different approaches for CF gene therapy; all of which ultimately have to be tested in preclinical model systems. Primary human nasal epithelial cultures (HNECs) derived from nasal turbinate brushing were used to test the efficiency of a helper-dependent adenoviral (HD-Ad) vector expressing CFTR. HD-Ad-CFTR transduction resulted in functional expression of CFTR at the apical membrane in nasal epithelial cells obtained from CF patients. These results suggest that HNECs can be used for preclinical testing of gene therapy vectors in CF.
نوع الوثيقة: article
وصف الملف: electronic resource
اللغة: English
تدمد: 2329-0501
Relation: http://www.sciencedirect.com/science/article/pii/S2329050116300468; https://doaj.org/toc/2329-0501
DOI: 10.1038/mtm.2015.34
URL الوصول: https://doaj.org/article/98027c0e7a7e4df392f9c3f70e156406
رقم الانضمام: edsdoj.98027c0e7a7e4df392f9c3f70e156406
قاعدة البيانات: Directory of Open Access Journals
الوصف
تدمد:23290501
DOI:10.1038/mtm.2015.34