Phenylalkenoic Acid-Mediated Corection of Aberant Protein Trafficking

This discovery encompasses the compound 4-phenyl-delta3-transbutenoic acid, which restores normal biosynthetic trafficking to the CFTR mutation deltaF508 The mechanism centers on butyrate-mediated down-regulation of the chaperone protein Hsc70. The compound 4-phenyl-delta3-transbutenoic acid is related to the compound biphenyl, which is currently approved for inherited urea cycle disorders where it functions to scavenge excess ammonia and has exhibited little toxicity.

Inventor(s): Brusilow, Saul W.

Type of Offer: Licensing



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