Cystic fibrosis in the era of precision medicine

Shruti M Paranjape; Peter J Mogayzel Jr

doi:10.1016/j.prrv.2017.03.001

Cystic fibrosis in the era of precision medicine

Paediatr Respir Rev. 2018 Jan:25:64-72. doi: 10.1016/j.prrv.2017.03.001. Epub 2017 Mar 9.

Authors

Shruti M Paranjape¹, Peter J Mogayzel Jr²

Affiliations

¹ Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins Medical Institutions, Baltimore, Maryland, USA. Electronic address: sparanj1@jhmi.edu.
² Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins Medical Institutions, Baltimore, Maryland, USA.

PMID: 28372929
DOI: 10.1016/j.prrv.2017.03.001

Abstract

The treatment of people with cystic fibrosis (CF) has been transformed by the availability of drugs that target the basic chloride defect in the disease. The use of drugs that target specific molecular defects embodies the goals of precision medicine, which incorporate preventive and therapeutic strategies and takes into account differences among individuals. However, the entirety of CF care, from diagnosis to understanding the clinical phenotype and developing a therapeutic strategy, depends on taking into account individual characteristics to achieve optimal outcomes. Future therapies are likely to be even more individualized ushering in a new era of precision medicine.

Keywords: CFTR; Cystic Fibrosis; Genotype; Phenotype; Precision Medicine.

Publication types

Review

MeSH terms

Cystic Fibrosis* / genetics
Cystic Fibrosis* / therapy
Humans
Patient Care Management* / methods
Patient Care Management* / trends
Pharmacogenetics
Precision Medicine / methods*
Preventive Health Services