ArticleGene therapy2007
Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice.
Article in Gene therapy, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
19 citing papers in PubMed, 35 citations in OpenAlex.
- Article
- Current landscape of cystic fibrosis gene therapy.Frontiers in pharmacology · 2024Review
- Antisense Oligonucleotide Therapeutics for Cystic Fibrosis: Recent Developments and Perspectives.Molecules and cells · 2023Review
- Cystic Fibrosis and the Cells of the Airway Epithelium: What Are Ionocytes and What Do They Do?Annual review of pathology · 2022Review
- Tissue and cell-type-specific transduction using rAAV vectors in lung diseases.Journal of molecular medicine (Berlin, Germany) · 2021Review
- Secretory Cells Dominate Airway CFTR Expression and Function in Human Airway Superficial Epithelia.American journal of respiratory and critical care medicine · 2021Article
- Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease.Human gene therapy · 2020Review
- Article
- Article
- Loss of Cftr function exacerbates the phenotype of Na(+) hyperabsorption in murine airways.American journal of physiology. Lung cellular and molecular physiology · 2013Article
- Transgenic hCFTR expression fails to correct β-ENaC mouse lung disease.American journal of physiology. Lung cellular and molecular physiology · 2012Article
- Clinical review: gene-based therapies for ALI/ARDS: where are we now?Critical care (London, England) · 2011Review
- Decreased soluble adenylyl cyclase activity in cystic fibrosis is related to defective apical bicarbonate exchange and affects ciliary beat frequency regulation.The Journal of biological chemistry · 2010Article
- Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.Molecular therapy : the journal of the American Society of Gene Therapy · 2010Article
- The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways.Biomaterials · 2010Article
- CFTR delivery to 25% of surface epithelial cells restores normal rates of mucus transport to human cystic fibrosis airway epithelium.PLoS biology · 2009Article
- Ion transport across CF and normal murine olfactory and ciliated epithelium.American journal of physiology. Cell physiology · 2009Article
- Influence of the cystic fibrosis transmembrane conductance regulator on expression of lipid metabolism-related genes in dendritic cells.Respiratory research · 2009Article
- The epithelial cholinergic system of the airways.Histochemistry and cell biology · 2008Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Successful gene therapy will require that the therapeutic gene be expressed at a sufficient level in the correct cell type(s). To improve the specificity of gene transfer for cystic fibrosis (CF) and other airway diseases, we have begun to develop cell-type specific promoters to target the expression of transgenes to specific airway cell types. Using a FOXJ1 promoter construct previously shown to direct transgene expression specifically to ciliated cells, we have generated transgenic mice expressing human cystic fibrosis transmembrane conductance regulator (CFTR) in the murine tracheal and nasal epithelia. RNA analysis demonstrated levels of CFTR expression is greater than or equal to the level of endogenous mouse CFTR. Immunoprecipitation and western blotting demonstrated the production of human CFTR protein, and immunochemistry confirmed that CFTR was expressed in the apical region of ciliated cells. The transgenic animals were bred to CFTR null mice (Cftr(tm1Unc)) to determine if expression of CFTR from the FOXJ1 promoter is capable of correcting the airway defects in Cl(-) secretion and Na(+) absorption that accompany CF. Isolated trachea from neonatal CF mice expressing the FOXJ1/CFTR transgene demonstrated a correction of forskolin-stimulated Cl(-) secretion. However, expression of human CFTR in ciliated cells of the nasal epithelia failed to significantly change the nasal bioelectrics of the CF mice.
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What OpenQuestion holds
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.