Journal article
New Directions in Pulmonary Gene Therapy
Human gene therapy, Vol.31(17-18), pp.921-939
09/01/2020
DOI: 10.1089/hum.2020.166
PMCID: PMC7495918
PMID: 32814451
Abstract
The lung has long been a target for gene therapy, yet efficient delivery and phenotypic disease correction has remained challenging. Although there have been significant advancements in gene therapies of other organs, including the development of severalex vivotherapies,in vivotherapeutics of the lung have been slower to transition to the clinic. Within the past few years, the field has witnessed an explosion in the development of new gene addition and gene editing strategies for the treatment of monogenic disorders. In this review, we will summarize current developments in gene therapy for cystic fibrosis, alpha-1 antitrypsin deficiency, and surfactant protein deficiencies. We will explore the different gene addition and gene editing strategies under investigation and review the challenges of delivery to the lung.
Details
- Title: Subtitle
- New Directions in Pulmonary Gene Therapy
- Creators
- Amber Vu - University of IowaPaul B. McCray - University of Iowa
- Resource Type
- Journal article
- Publication Details
- Human gene therapy, Vol.31(17-18), pp.921-939
- Publisher
- Mary Ann Liebert, Inc
- DOI
- 10.1089/hum.2020.166
- PMID
- 32814451
- PMCID
- PMC7495918
- ISSN
- 1043-0342
- eISSN
- 1557-7422
- Number of pages
- 19
- Grant note
- P01 HL091842; P01 HL152960; UG3 HL147366 / NHLBI NIH HHS; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Heart Lung & Blood Institute (NHLBI) T32 AI007343 / NIAID NIH HHS; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Allergy & Infectious Diseases (NIAID) P30 DK054759 / NIDDK NIH HHS; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK)
- Language
- English
- Date published
- 09/01/2020
- Academic Unit
- Microbiology and Immunology; Pulmonary Medicine; Stead Family Department of Pediatrics; Internal Medicine
- Record Identifier
- 9984297428302771
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