Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Somayaji, Ranjani; Nichols, Dave P; Bell, Scott C

doi:10.1080/13543784.2020.1805733

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Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

Somayaji, Ranjani, Nichols, Dave P, Bell, Scott C·2020·Expert Opinion on Investigational Drugs

DOI 10.1080/13543784.2020.1805733

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Abstract

Introduction Cystic fibrosis (CF) is a genetic disease affecting multiple organ systems. Research and innovations in novel therapeutic agents and health care delivery have resulted in dramatic improvements in quality of life and survival for people with CF. Despite this, significant disease burden persists for many and this is compounded by disparities in treatment access and care which globally necessitates further work to improve outcomes. Because of the advent of numerous therapies which include gene-targeted modulators in parallel with specialized care delivery models, innovative efforts continue. Areas covered In this review, we discuss the available data on investigational agents in clinical development and currently available treatments for CF. We also evaluate approaches to care delivery, consider treatment gaps, and propose future directions for advancement. Expert opinion Since the discovery of the CF gene, CFTR modulators have provided a hallmark of success, even though it was thought not previously possible. This has led to reinvigorated efforts and innovations in treatment approaches and care delivery. Numerous challenges remain because of genetic and phenotypic heterogeneity, access issues, and therapeutic costs, but the collaborative approach between stakeholders for continued innovation fuels optimism. Abbreviations: CF cystic fibrosis; CFF Cystic Fibrosis Foundation (USA); CFTR cystic fibrosis transmembrane regulator; CRISPR clustered regularly interspaced sh…

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Sorted by citations

Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing
Mengstie, M. A., & Wondimu, B. Z. (2021) · Biologics: Targets and Therapy · doi:10.2147/btt.s326422 · Cited by 478

See all 12

Authors: Somayaji, R., Nichols, D. P., & Bell, S. C.
Affiliations: Departments of Medicine; Microbiology, Immunology & Infectious Disease; Community Health Sciences, University of Calgary, Calgary, AB, Canada
Department of Pediatrics, Seattle Children’s Hospital, Seattle, WA, USA
Department of Thoracic Medicine, The Prince Charles Hospital, Brisbane, QLD, Australia
ORCID: Somayaji, Ranjani
Nichols, Dave P
Bell, Scott C
Venue: Expert Opinion on Investigational Drugs
Publisher: Informa UK Limited
Published: 2020-10-02
Volume: 29
Issue: 10
Pages: 1107-1124
ISSN: 1354-3784
1744-7658
DOI: 10.1080/13543784.2020.1805733
Cited by: 12
References: 12
Language: en

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The paper: Somayaji, R., Nichols, D. P., & Bell, S. C. (2020). Cystic fibrosis – Ten promising therapeutic approaches in the current era of care. Expert Opinion on Investigational Drugs. https://doi.org/10.1080/13543784.2020.1805733

Cystic fibrosis – Ten promising therapeutic approaches in the current era of care

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