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PS-341 Rescues Cystic Fibrosis Transmembrane Regulator (CFTR) from Endoplasmic Reticulum Mediated Degradation

UNMET NEEDThere is understanding that mutations in the cystic fibrosis transmembrane regulator (CFTR) protein can lead to life-threatening illness. When functioning normally, CFTR is thought to be a necessary cAMP-activated chloride channel. In CF, this channel is thought to be misprocessed and retained in the endoplasmic reticulum of epithelial cells,...

Published: 3/13/2025 | Inventor(s): Neeraj Vij, Pamela Zeitlin

Keywords(s): Biologics, Cystic Fibrosis, Cystic Fibrosis (CF), Disease Indication, Genetic Disorders, Protein, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Genetic Diseases > Cystic Fibrosis, Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Respiratory Diseases, Technology Classifications > Therapeutic Modalities > Proteins, Technology Classifications > Therapeutic Modalities > Small Molecules, Clinical and Disease Specializations > Genetic Diseases, Technology Classifications > Therapeutic Modalities