ArticleThe EMBO journal1997
Complementation of null CF mice with a human CFTR YAC transgene.
Article in The EMBO journal, 1997. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
26 citing papers in PubMed.
- On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis.Frontiers in pharmacology · 2021Review
- A BAC Transgene Expressing Human CFTR under Control of Its Regulatory Elements Rescues Cftr Knockout Mice.Scientific reports · 2019Article
- Animal models for cystic fibrosis liver disease (CFLD).Biochimica et biophysica acta. Molecular basis of disease · 2019Review
- RNA Splicing and Disease: Animal Models to Therapies.Trends in genetics : TIG · 2019Review
- Animal and model systems for studying cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2018Review
- Nanoparticle-mediated rhodopsin cDNA but not intron-containing DNA delivery causes transgene silencing in a rhodopsin knockout model.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2016Article
- Physiological impact of abnormal lipoxin A₄ production on cystic fibrosis airway epithelium and therapeutic potential.BioMed research international · 2015Review
- Characterization of a large human transgene following invasin-mediated delivery in a bacterial artificial chromosome.Chromosoma · 2013Article
- Regulated traffic of anion transporters in mammalian Brunner's glands: a role for water and fluid transport.American journal of physiology. Gastrointestinal and liver physiology · 2013Article
- CFTR, mucins, and mucus obstruction in cystic fibrosis.Cold Spring Harbor perspectives in medicine · 2012Review
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- Article
- Transgene design.Methods in molecular biology (Clifton, N.J.) · 2011Article
- CFTR expression and activity from the human CFTR locus in BAC vectors, with regulatory regions, isolated by a single-step procedure.Gene therapy · 2010Article
- Prospects for the use of artificial chromosomes and minichromosome-like episomes in gene therapy.Journal of biomedicine & biotechnology · 2010Article
- CFTR expression from a BAC carrying the complete human gene and associated regulatory elements.Journal of cellular and molecular medicine · 2009Article
- Generation of a conditional null allele for Cftr in mice.Genesis (New York, N.Y. : 2000) · 2008Article
- Transcription-dependent spatial arrangements of CFTR and conserved adjacent loci are not conserved in human and murine nuclei.Chromosoma · 2008Article
- A transgenic mouse model engineered to investigate human brain-derived neurotrophic factor in vivo.Transgenic research · 2007Article
- A new method for generating point mutations in bacterial artificial chromosomes by homologous recombination in Escherichia coli.Nucleic acids research · 2001Article
Corrections and comments
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Authors and funding
11 authors.
Funding
Abstract
We have made transgenic mice carrying a 320 kb YAC with the intact human cystic fibrosis transmembrane regulator (CFTR) gene. Mice that only express the human transgene were obtained by breeding with Cambridge null CF mice. One line has approximately two copies of the intact YAC. Mice carrying this transgene and expressing no mouse cftr appear normal and breed well, in marked contrast to the null mice, where 50% die by approximately 5 days after birth. The chloride secretory responses in these mice are as large or larger than in wild-type tissues. Expression of the transgene is highly cell type specific and matches that of the endogenous mouse gene in the crypt epithelia throughout the gut and in salivary gland tissue. However, there is no transgene expression in some tissues, such as the Brunner's glands, where it would be expected. Where there are differences between the mouse and human pattern of expression, the transgene follows the mouse pattern. We have thus defined a cloned fragment of DNA which directs physiological levels of expression in many of the specific cells where CFTR is normally expressed.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.