Use of protein repair therapy in the treatment of cystic fibrosis

Ronald C. Rubenstein, Pamela L. Zeitlin

Research output: Contribution to journalReview articlepeer-review

Abstract

Disruption in the biosynthesis or function the cystic fibrosis transmembrane conductance regulator (CFTR) results from over 700 different mutations in the CFTR gene. It is useful to classify these mutations by the nature of the resulting defect. Understanding the molecular mechanism that leads to CFTR dysfunction stimulates the design of therapeutic strategies based on restoration of CFTR function to the mutant protein, or 'protein repair therapy.' This review links the classification of CFTR mutations to a number of new pharmacologic strategies that lead to enhancement of CFTR function by manipulation of mutant CFTR.

Original languageEnglish (US)
Pages (from-to)250-255
Number of pages6
JournalCurrent opinion in pediatrics
Volume10
Issue number3
DOIs
StatePublished - 1998

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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