Abstract

A REVIEW ON CURRENT THERAPEUTIC TARGETS IN CYSTIC FIBROSIS

Lijolin V. S.*, Devika J. J., Prasobh G. R. and Grace N. Raju

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Abstract

Cystic fibrosis is an autosomal recessive disorder caused by mutations in the CFTR gene that lead to abnormalities in the chloride channels in the cells that produce mucus and sweat. The respiratory system and GIT are mainly involved but also many organs are affected and lead to life threatening complication. Management requires drug therapy comprehensive physiotheraphy and nutritional support. Previously,the focus was on symptoms improvement and complication prevention but recently protein rectifers have been studied that are said to correct basic structural and functional abnormalities. Some improvement is seen with corrector medications. Other promising approaches include gene therapy, targeting cellular interactions and new drugs for symptomatic improvement in individuals with CF.

Keywords: Mutation, respiratory, CFTR, gene therapy.