Cited by 36Virus-based gene therapy researchRNA Interference and Gene DeliveryInhalation and Respiratory Drug Delivery

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Sondhi, Dolan, Stiles, Katie M., De, Bishnu P., Crystal, Ronald G.·2017·Human Gene Therapy

Abstract

Genetic modification therapy is a promising therapeutic strategy for many diseases of the lung intractable to other treatments. Lung gene therapy has been the subject of numerous preclinical animal experiments and human clinical trials, for targets including genetic diseases such as cystic fibrosis and α1-antitrypsin deficiency, complex disorders such as asthma, allergy, and lung cancer, infections such as respiratory syncytial virus (RSV) and Pseudomonas , as well as pulmonary arterial hypertension, transplant rejection, and lung injury. A variety of viral and non-viral vectors have been employed to overcome the many physical barriers to gene transfer imposed by lung anatomy and natural defenses. Beyond the treatment of lung diseases, the lung has the potential to be used as a metabolic factory for generating proteins for delivery to the circulation for treatment of systemic diseases. Although much has been learned through a myriad of experiments about the development of genetic modification of the lung, more work is still needed to improve the delivery vehicles and to overcome challenges such as entry barriers, persistent expression, specific cell targeting, and circumventing host anti-vector responses.

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Crossref · 10.1089/hum.2016.152

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609 of 609 references verified0 citation errors · 0 not checkable

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Authors
Sondhi, D., Stiles, K. M., De, B. P., & Crystal, R. G.
Affiliations
Weill Cornell Medicine (US)
Venue
Human Gene Therapy
Published
2017-01
Volume
28
Issue
1
Pages
3-84
ISSN
  • 1043-0342 (ISSN-L)
  • 1557-7422
OpenAlex
W2582514264
Cited by
36
References
609
Citations by year
2026: 1 · 2023: 3 · 2022: 7 · 2021: 4 · 2020: 9 · 2019: 2 · 2018: 6 · 2017: 4
FWCI
0.53
Type
journal article
Language
en
Open access
closed
Locations
  • Human Gene Therapy (publishedVersion)
  • PubMed (publishedVersion)
Topics
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
  • Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Inhalation and Respiratory Drug Delivery
Keywords
pulmonary gene therapy, viral vectors, non-viral vectors, cystic fibrosis, alpha-1 antitrypsin deficiency, asthma, allergy, lung cancer, respiratory syncytial virus, Pseudomonas infection, pulmonary arterial hypertension, graft rejection, lung injury, persistent gene expression, cell targeting
MeSH terms
Animals, Genetic Vectors, Humans, Lung Diseases, Viruses, Genetic Therapy

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