Phenotypic Characterization and Comparison of Cystic Fibrosis Rat Models Generated Using CRISPR/Cas9 Gene Editing
利用CRISPR / Cas9基因编辑产生囊性纤维化大鼠模型的表型特征及比较
作者: Alexandra McCarronPatricia CmielewskiNicole ReyneChantelle McIntyreJohn FinnieFiona CraigNathan Rout-PittJuliette DelhoveJohn E. SchjenkenHon Y. ChanBernadette BoogEmma KnightRodney C. GilmoreWanda K. O'NealRichard C. BoucherDavid ParsonsMartin Donnelley
作者单位: 1Department of Respiratory and Sleep Medicine, Women's and Children's Hospital, North Adelaide, South Australia, Australia
2Adelaide Medical School, University of Adelaide, Adelaide, South Australia, Australia
3Robinson Research Institute, University of Adelaide, Adelaide, South Australia, Australia
4Genetics and Molecular Pathology, SA Pathology at Women's and Children's Hospital, North Adelaide, South Australia, Australia
5Division of Anatomical Pathology, SA Pathology, Adelaide, South Australia, Australia
6Marsico Lung Institute/Cystic Fibrosis Research Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina
刊名: The American Journal of Pathology, 2020, Vol.190 (5), pp.977-993
来源数据库: Elsevier Journal
DOI: 10.1016/j.ajpath.2020.01.009
英文摘要: Animal models of cystic fibrosis (CF) are essential for investigating disease mechanisms and trialing potential therapeutics. This study generated two CF rat models using clustered regularly interspaced short palindromic repeats/clustered regularly interspaced short palindromic repeats–associated protein 9 gene editing. One rat model carries the common human Phe508del (ΔF508) CF transmembrane conductance regulator ( CFTR ) mutation, whereas the second is a CFTR knockout model. Phenotype was characterized using a range of functional and histologic assessments, including nasal potential difference to measure electrophysiological function in the upper airways, RNAscope in situ hybridization and quantitative PCR to assess CFTR mRNA expression in the lungs, immunohistochemistry to localize...
全文获取路径: Elsevier  (合作)
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影响因子:4.522 (2012)

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