COMPOSITIONS AND METHODS FOR ENHANCING ION TRANSPORTER ACTIVITY AND USES THEREOF

Methods for enhancing the activity of an ion transporter in a cell and/or treating a channelopathy in a subject based on the administration or use of an effective amount of a fenretinide compound (fenretinide, a fenretinide analog, or a pharmaceutically acceptable salt thereof) and/or zinc are descr...

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Bibliographic Details
Main Authors ABU-ARISH, Asmahan, GARIC, Dusan, HANRAHAN, John, RADZIOCH, Danuta
Format Patent
LanguageEnglish
Published 08.04.2021
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:Methods for enhancing the activity of an ion transporter in a cell and/or treating a channelopathy in a subject based on the administration or use of an effective amount of a fenretinide compound (fenretinide, a fenretinide analog, or a pharmaceutically acceptable salt thereof) and/or zinc are described. The methods are useful for restoring or increasing the cell surface expression of an ion transporter such as the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) that is mutated or defective. The fenretinide compound and/or zinc may be used in combination with ion transporter modulators, e.g., CFTR modulators, to further enhance the cell surface expression and activity of the ion transporter.
Bibliography:Application Number: US201817047678