The DOI matches its Crossref record; 12 of the 12 references it cites were found in a registry or an index.
Cystic fibrosis – Ten promising therapeutic approaches in the current era of care
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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“Although there is no cure for cystic fibrosis, symptom-based therapies (such as antibiotics, bronchodilators, and mucus thinning medications) and CFTR modulating drugs have become the first-line treatments to relieve symptoms and reduce the risk of complications.31”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [31]
In the citing paper
“Although there is no cure for cystic fibrosis, symptom-based therapies (such as antibiotics, bronchodilators, and mucus thinning medications) and CFTR modulating drugs have become the first-line treatments to relieve symptoms and reduce the risk of complications.31”
From Mechanism and Applications of CRISPR/Cas-9-Mediated Genome Editing (2021) · cites this paper as [31]
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Cited by
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
Metadata
- 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
- 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
- Cited by
- 12
- References
- 12
- Language
- en
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