The UAB Research Foundation (The University of Alabama at Birmingham)

The General Hospital Corporation

Southern Research Institute

Steven Mark Rowe

Guillermo J Tearney

Brett Eugene Bouma

Eric J Sorscher

Linbo Liu

Kengyeh Ken Chu

David M Bedwell

Bradford James Diephuis

Eric James Wilsterman

Gregory Andrew Dierksen

Corinne E Augelli-Szafran

Bini Mathew

Kim M Keeling

John Phillip Tillotson

Ming Du

Mark Dransfield

Sammeta Vamsee Raju

Terry Smalley

Kevin Jay Rodzinak

Lawrence Rasmussen

Robert Hunter

Venkateshwar Mutyam

Eric James Wilsterman Jr

Gregory Andrew Dirksen

The present disclosure is concerned with substituted uracil compounds, pharmaceutical compositions comprising the compounds, and methods of treating disorders associated with the presence of a premature termination codon such as, for example, cystic fibrosis, Duchenne muscular dystrophy, aniridia, Becker muscular dystrophy, spinal muscular atrophy, Hurler syndrome, hemophilia, epidermolysis bullosa (e.g., dystrophic (DEB) form, junctional (JEB) form), Usher syndrome, and cancer, using the compounds. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Uracil derivatives for stimulating read-through of premature termination codons

Provided herein are methods of increasing mucus clearance in a subject having decreased mucus clearance. Also provided are methods of treating or preventing a respiratory disorder.

Methods and compositions for increasing mucus clearance

The present disclosure provides compositions and methods for treating and/preventing diseases and conditions associated with premature termination mutations. The methods disclosed herein comprise the step of administering to the subject a therapeutically effective dose of a compound described herein that induces read-through of the premature termination codon. The methods of the present disclosure may further comprise treating the subject with enzyme replacement therapy wherein the enzyme replacement therapy is selected for the disease or condition to be treated. Furthermore, the present disclosure provides method of pharmacologically suppressing premature termination codons in a subject in need of such suppression.

Methods and compounds for stimulating read-through of premature termination codons

Exemplary apparatus and method can be provided for obtaining data regarding a plurality of samples. For example, using at least one arrangement, it is possible to receive interferometric information that is based on radiations provided from a reference and the samples that are provided in respective chambers. Alternatively and/or in addition, based on the interferometric information, it is possible to discriminate between agents to identify a particular agent that affects a particular function within at least one of the samples.

Methods, systems, arrangements and computer-accessible medium for providing micro-optical coherence tomography procedures

The present disclosure provides compounds effective in increasing mucociliary clearance in a subject. In one embodiment, the compounds are of the general formula I. The present disclosure further shows that such compounds are effective in increasing activation of the CFTR, thereby increasing mucociliary clearance in the subject. The present disclosure further shows that such compounds are effective in increasing the depth of ASL, thereby increasing mucociliary clearance in the subject. In one embodiment of each of the foregoing, the subject is free from congenital or genetic defect in the cellular mucociliary clearance apparatus and/or acquired abnormality in the cellular mucociliary clearance apparatus.

Methods for increasing CFTR activity

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Birmingham, AL, United States of America

Steven Mark Rowe