Amphiphilic shuttle peptide delivers base editor ribonucleoprotein to correct the CFTR R553X mutation in well-differentiated airway epithelial cells.

Autor: Kulhankova K; Department of Pediatrics, University of Iowa, Iowa City, IA, USA., Cheng AX; Feldan Therapeutics, Quebec, Qc, Canada., Traore S; Department of Pediatrics, University of Iowa, Iowa City, IA, USA., Auger M; Feldan Therapeutics, Quebec, Qc, Canada.; Department of Chemical Engineering, Laval University, Quebec, Qc, Canada., Pelletier M; Feldan Therapeutics, Quebec, Qc, Canada.; Department of Chemical Engineering, Laval University, Quebec, Qc, Canada., Hervault M; Feldan Therapeutics, Quebec, Qc, Canada., Wells KD; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Green JA; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Byrne A; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Nelson B; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Sponchiado M; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Boosani C; Division of Animal Sciences, Swine Somatic Cell Genome Editing Center, University of Missouri, Columbia, MO, USA., Heffner CS; The Jackson Laboratory, Genetic Resource Science, Bar Harbor, ME, USA., Snow KJ; The Jackson Laboratory, Genetic Resource Science, Bar Harbor, ME, USA., Murray SA; The Jackson Laboratory, Genetic Resource Science, Bar Harbor, ME, USA., Villacreses RA; Department of Internal Medicine, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Rector MV; Department of Internal Medicine, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA, USA.; Pappajohn Biomedical Institute, University of Iowa, Iowa City, IA, USA., Gansemer ND; Department of Internal Medicine, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA, USA.; Pappajohn Biomedical Institute, University of Iowa, Iowa City, IA, USA., Stoltz DA; Department of Internal Medicine, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA, USA.; Pappajohn Biomedical Institute, University of Iowa, Iowa City, IA, USA.; Department of Biomedical Engineering, University of Iowa, Iowa City, IA, USA.; Department of Molecular Physiology and Biophysics, Roy J. and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, IA, USA., Allamargot C; Central Microscopy Research Facility (CMRF), and Office for the Vice President of Research (OVPR), University of Iowa, Iowa City, IA, USA., Couture F; TransBIOTech, Lévis, Qc, Canada., Hemez C; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.; Graduate Program in Biophysics, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Hallée S; Feldan Therapeutics, Quebec, Qc, Canada., Barbeau X; Feldan Therapeutics, Quebec, Qc, Canada., Harvey M; Feldan Therapeutics, Quebec, Qc, Canada., Lauvaux C; Feldan Therapeutics, Quebec, Qc, Canada., Gaillet B; Department of Chemical Engineering, Laval University, Quebec, Qc, Canada., Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA., Liu DR; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA., McCray PB Jr; Department of Pediatrics, University of Iowa, Iowa City, IA, USA., Guay D; Feldan Therapeutics, Quebec, Qc, Canada.; Department of Chemical Engineering, Laval University, Quebec, Qc, Canada.

Publikováno v: Nucleic acids research [Nucleic Acids Res] 2024 Oct 28; Vol. 52 (19), pp. 11911-11925.

Cystic Fibrosis Transmembrane Conductance Regulator in Sarcoplasmic Reticulum of Airway Smooth Muscle. Implications for Airway Contractility.

Autor: Cook DP; 1 Department of Internal Medicine.; 2 Department of Molecular Physiology and Biophysics., Rector MV; 1 Department of Internal Medicine., Bouzek DC; 1 Department of Internal Medicine., Michalski AS; 1 Department of Internal Medicine., Gansemer ND; 1 Department of Internal Medicine., Reznikov LR; 1 Department of Internal Medicine., Li X; 1 Department of Internal Medicine., Stroik MR; 1 Department of Internal Medicine., Ostedgaard LS; 1 Department of Internal Medicine., Abou Alaiwa MH; 1 Department of Internal Medicine., Thompson MA; 3 Department of Anesthesiology and.; 4 Department of Physiology and Biomedical Engineering, Mayo Clinic, Rochester, Minnesota., Prakash YS; 3 Department of Anesthesiology and.; 4 Department of Physiology and Biomedical Engineering, Mayo Clinic, Rochester, Minnesota., Krishnan R; 5 Department of Emergency Medicine, Center for Vascular Biology Research, Beth Israel Deaconess Medical Center, Boston, Massachusetts; and., Meyerholz DK; 6 Department of Pathology., Seow CY; 7 Department of Pathology and Laboratory Medicine, James Hogg Research Centre/St. Paul's Hospital, University of British Columbia, Vancouver, British Columbia, Canada., Stoltz DA; 1 Department of Internal Medicine.; 2 Department of Molecular Physiology and Biophysics.; 8 Department of Biomedical Engineering, and.; 9 Pappajohn Biomedical Institute, University of Iowa, Iowa City, Iowa.

Publikováno v: American journal of respiratory and critical care medicine [Am J Respir Crit Care Med] 2016 Feb 15; Vol. 193 (4), pp. 417-26.

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs.

Autor: Stoltz DA; Department of Internal Medicine, University of Iowa Roy J. and Lucille A. Carver College of Medicine, Iowa City, Iowa 52242, USA. michael-welsh@uiowa.edu, Rokhlina T, Ernst SE, Pezzulo AA, Ostedgaard LS, Karp PH, Samuel MS, Reznikov LR, Rector MV, Gansemer ND, Bouzek DC, Abou Alaiwa MH, Hoegger MJ, Ludwig PS, Taft PJ, Wallen TJ, Wohlford-Lenane C, McMenimen JD, Chen JH, Bogan KL, Adam RJ, Hornick EE, Nelson GA 4th, Hoffman EA, Chang EH, Zabner J, McCray PB Jr, Prather RS, Meyerholz DK, Welsh MJ

Publikováno v: The Journal of clinical investigation [J Clin Invest] 2013 Jun; Vol. 123 (6), pp. 2685-93. Date of Electronic Publication: 2013 May 08.

Expression of human paraoxonase 1 decreases superoxide levels and alters bacterial colonization in the gut of Drosophila melanogaster.

Autor: Pezzulo AA; Department of Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, Iowa, United States of America., Hornick EE, Rector MV, Estin M, Reisetter AC, Taft PJ, Butcher SC, Carter AB, Manak JR, Stoltz DA, Zabner J

Publikováno v: PloS one [PLoS One] 2012; Vol. 7 (8), pp. e43777. Date of Electronic Publication: 2012 Aug 30.

Loss of anion transport without increased sodium absorption characterizes newborn porcine cystic fibrosis airway epithelia.

Autor: Chen JH; Department of Internal Medicine, University of Iowa, Iowa City, 52242, USA., Stoltz DA, Karp PH, Ernst SE, Pezzulo AA, Moninger TO, Rector MV, Reznikov LR, Launspach JL, Chaloner K, Zabner J, Welsh MJ

Publikováno v: Cell [Cell] 2010 Dec 10; Vol. 143 (6), pp. 911-23.