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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
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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
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Clinical and Disease Specializations > Genetic Diseases > Cystic Fibrosis
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Clinical and Disease Specializations > Rare Diseases
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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
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