The effect of premature termination codon mutations on CFTR mRNA abundance in human nasal epithelium and intestinal organoids: a basis for read‐through therapies in cystic fibrosis

Nonsense-mediated mRNA decay: a ‘nonsense’ pathway makes sense in stem cell biology

(2017) Xin Han et al.   NUCLEIC ACIDS RESEARCH

New Pharmacological Approaches to Treat Patients with Cystic Fibrosis with Nonsense Mutations

(2016) Isabelle Sermet-Gaudelus et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Airway Progenitor Clone Formation Is Enhanced by Y-27632–Dependent Changes in the Transcriptome

(2016) Susan D. Reynolds et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

mRNA-based detection of rare CFTR mutations improves genetic diagnosis of cystic fibrosis in populations with high genetic heterogeneity

(2016) V. Felício et al.   CLINICAL GENETICS

Limited premature termination codon suppression by read-through agents in cystic fibrosis intestinal organoids

(2016) D.D. Zomer-van Ommen et al.   Journal of Cystic Fibrosis

From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations

(2016) Gudio Veit et al.   MOLECULAR BIOLOGY OF THE CELL

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

(2016) Johanna F. Dekkers et al.   Science Translational Medicine

Progress in therapies for cystic fibrosis

(2016) Kris De Boeck et al.   Lancet Respiratory Medicine

Nonsense-Mediated RNA Decay Influences Human Embryonic Stem Cell Fate

(2016) Chih-Hong Lou et al.   Stem Cell Reports

Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis

(2015) Nikhil T. Awatade et al.   EBioMedicine

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Growing Self-Organizing Mini-Guts from a Single Intestinal Stem Cell: Mechanism and Applications

(2013) T. Sato et al.   SCIENCE

Suppression of premature termination codons as a therapeutic approach

(2012) Kim M. Keeling et al.   CRITICAL REVIEWS IN BIOCHEMISTRY AND MOLECULAR BIOLOGY

Primer3—new capabilities and interfaces

(2012) Andreas Untergasser et al.   NUCLEIC ACIDS RESEARCH

Long-term Expansion of Epithelial Organoids From Human Colon, Adenoma, Adenocarcinoma, and Barrett's Epithelium

(2011) Toshiro Sato et al.   GASTROENTEROLOGY

The Unfolded Protein Response: From Stress Pathway to Homeostatic Regulation

(2011) P. Walter et al.   SCIENCE

Deletion of CFTR Translation Start Site Reveals Functional Isoforms of the Protein in CF Patients

(2009) Anabela S. Ramalho et al.   CELLULAR PHYSIOLOGY AND BIOCHEMISTRY

The CFTR frameshift mutation 3905insT and its effect at transcript and protein level

(2009) Javier Sanz et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Single Lgr5 stem cells build crypt-villus structures in vitro without a mesenchymal niche

(2009) Toshiro Sato et al.   NATURE