Will airway gene therapy for cystic fibrosis improve lung function? New imaging technologies can help us find out
| dc.contributor.author | Parsons, D. | |
| dc.contributor.author | Donnelley, M. | |
| dc.date.issued | 2020 | |
| dc.description.abstract | The promise of genetic therapies has turned into reality in recent years, with new first-line treatments for fatal diseases now available to patients. The development and testing of genetic therapies for respiratory diseases such as cystic fibrosis (CF) has also progressed. The addition of gene editing to the genetic agent toolbox, and its early success in other organ systems, suggests we will see rapid expansion of gene correction options for CF in the future. Although substantial progress has been made in creating techniques and genetic agents that can be highly effective for CF correction in vitro, physiologically relevant functional in vivo changes have been largely prevented by poor delivery efficiency within the lungs. Somewhat hidden from view, however, is the absence of reliable, accurate, detailed, and noninvasive outcome measures that can detect subtle disease and treatment effects in the lungs of humans or animal models. The ability to measure the fundamental function of the lung—ventilation, the effective transport of air throughout the lung—has been constrained by the available measurement technologies. Without sensitive measurement methods, it is difficult to quantify the effectiveness of genetic therapies for CF. The mainstays of lung health assessment are spirometry, which cannot provide adequate disease localization and is not sensitive enough to detect small early changes in disease; and computed tomography, which provides structural rather than functional information. Magnetic resonance imaging using hyperpolarized gases is increasingly useful for lung ventilation assessment, and it removes the radiation risk that accompanies X-ray methods. A new lung imaging technique, X-ray velocimetry, can now offer highly detailed regional lung ventilation information well suited to the diagnosis, treatment, and monitoring needs of CF lung disease, particularly after the application of genetic therapies. In this review, we discuss the options now available for imaging-based lung function measurement in the generation and use of genetic and other therapies for treating CF lung disease. | |
| dc.description.statementofresponsibility | David Parsons and Martin Donnelley | |
| dc.identifier.citation | Human Gene Therapy, 2020; 31(17-18):973-984 | |
| dc.identifier.doi | 10.1089/hum.2020.153 | |
| dc.identifier.issn | 1043-0342 | |
| dc.identifier.issn | 1557-7422 | |
| dc.identifier.orcid | Parsons, D. [0000-0002-8775-3501] [0000-0003-1746-3290] | |
| dc.identifier.orcid | Donnelley, M. [0000-0002-5320-7756] | |
| dc.identifier.uri | http://hdl.handle.net/2440/131775 | |
| dc.language.iso | en | |
| dc.publisher | Mary Ann Liebert | |
| dc.relation.grant | http://purl.org/au-research/grants/nhmrc/GNT1160011 | |
| dc.rights | © 2020 by Mary Ann Liebert, Inc. | |
| dc.source.uri | https://doi.org/10.1089/hum.2020.153 | |
| dc.subject | Gene therapy, cystic fibrosis; lung; lung function; imaging modalities; computed tomography; magnetic resonance imaging; hyperpolarized gas; X-ray velocimetry | |
| dc.title | Will airway gene therapy for cystic fibrosis improve lung function? New imaging technologies can help us find out | |
| dc.type | Journal article | |
| pubs.publication-status | Published |