A major problem with experiments involving the correction of cystic fibrosis by gene transfer has been reliably detecting the transgenic message and distinguishing this from an endogenous message and from vector DNA. We have exploited the SV40 small T antigen intron present in an expression vector containing the CFTR cDNA (pCFAS) using a primer directed to the spliced sequence to allow specific and precise detection of the transgeneic message.
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http://dx.doi.org/10.1038/sj.gt.3300469 | DOI Listing |
J Exp Zool A Ecol Integr Physiol
December 2024
Departement of Biology, Faculty of Science, Academic Assembly, University of Toyama, Gofuku, Toyama, Japan.
In euryhaline teleosts, the cystic fibrosis transmembrane conductance regulator (CFTR) in seawater (SW)-type chloride cells facilitates apical Cl secretion for SW adaptation, while alternative Cl excretion pathways remain understudied. This study investigates the role of the calcium-activated chloride channel, Anoctamin 1 (ANO1), in the gills of the euryhaline Japanese medaka (Oryzias latipes) under hyperosmolality and cortisol (CORT) influence. Acclimation to artificial SW, NaCl, mannitol, or glucose significantly upregulated ANO1 and CFTR mRNA expression in gills, unlike urea treatment.
View Article and Find Full Text PDFCurr Med Sci
December 2024
A V Pharma LLC, 1545 University Blvd N Ste A, Jacksonville, 32211, USA.
Cystic fibrosis (CF) is a hereditary disorder characterized by mutations in the CFTR gene, leading to impaired chloride ion transport and subsequent thickening of mucus in various organs, particularly the lungs. Despite significant progress in CF management, current treatments focus mainly on symptom relief and do not address the underlying genetic defects. Stem cell and gene therapies present promising avenues for tackling CF at its root cause.
View Article and Find Full Text PDFMicroorganisms
November 2024
Department of Bioscience, Silla University, Busan 46958, Republic of Korea.
This study aimed to observe the therapeutic effect of HFY11 (LP-HFY11) on lincomycin hydrochloride-induced diarrhea in mice. The results showed that LP-HFY11 alleviated weight loss and intestinal and colon tissue lesions caused by diarrhea. The serum assay showed that LP-HFY11 decreased interleukin 17A (IL-17A), IL-6, 5-hydroxytryptamine, and malondialdehyde levels and increased total antioxidant capacity in mice with diarrhea.
View Article and Find Full Text PDFNAR Mol Med
October 2024
Center for Genetic Diseases, Rosalind Franklin University of Medicine and Science, 3333 Green Bay Rd, North Chicago, IL 60064, USA.
J Cyst Fibros
November 2024
Department of Genetics and Genome Sciences, Case Western Reserve University, Cleveland, Ohio, United States. Electronic address:
Background: People with cystic fibrosis carrying two nonsense alleles lack CFTR-specific treatment. Growing evidence supports the hypothesis that nonsense mutation identity affects therapeutic response, calling for mutation-specific CF models. We describe a novel W1282X mouse model and compare it to an existing G542X mouse.
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