Nanomedicine for Cystic Fibrosis

Victor Ong; Vincent Mei; Lin Cao; Kiana Lee; Eun Ji Chung

doi:10.1177/2472630318824334

Nanomedicine for Cystic Fibrosis

SLAS Technol. 2019 Apr;24(2):169-180. doi: 10.1177/2472630318824334. Epub 2019 Feb 1.

Authors

Victor Ong¹, Vincent Mei¹, Lin Cao¹, Kiana Lee¹, Eun Ji Chung^{1

2

3

4

5

6}

Affiliations

¹ 1 Department of Biomedical Engineering, University of Southern California, Los Angeles, CA, USA.
² 2 Mork Family Department of Chemical Engineering and Materials Science, University of Southern California, Los Angeles, CA, USA.
³ 3 Division of Nephrology and Hypertension, Department of Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
⁴ 4 Division of Vascular Surgery and Endovascular Repair, Department of Surgery, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
⁵ 5 Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA, USA.
⁶ 6 Department of Stem Cell Biology and Regenerative Medicine, University of Southern California, Los Angeles, CA, USA.

PMID: 30707858
DOI: 10.1177/2472630318824334

Abstract

Cystic fibrosis is a genetic disease affecting more than 70,000 people worldwide. Caused by a mutation in the CFTR gene, cystic fibrosis can result in difficulty breathing, widespread bacterial infections, edema, malnutrition, pancreatitis, and death. Current drug-based treatments struggle to reach the site of action due to the thick mucus, and only manage symptoms such as blocked airways, lung infections, and limited ability to digest food. Nanotechnology opens up possibilities for improved treatment strategies by focusing on drug penetration through the mucus lining, eliminating resulting bacterial infections, and targeting the underlying genetic cause of the disease. In this review, we present recent nanoparticle developments for cystic fibrosis, challenges in nanomedicine therapeutics, and future research directions in gene editing and nonviral vectors for gene delivery.

Keywords: antibiotics; cystic fibrosis; gene therapy; mucus; nanobiotech; nanotechnology.

Publication types

Review

MeSH terms

Cystic Fibrosis / therapy*
Drug Carriers / administration & dosage*
Drug Delivery Systems / methods*
Genetic Therapy / methods*
Humans
Molecular Targeted Therapy / methods*
Nanomedicine / methods*
Nanoparticles / administration & dosage*

Substances

Drug Carriers