Genetic modification of the lung directed toward treatment of human disease

Dolan Sondhi, Katie M. Stiles, Bishnu P. De, Ronald Crystal

Research output: Contribution to journalReview article

10 Citations (Scopus)


Geneticmodification therapy isapromising therapeutic strategy formanydiseases of thelung 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 a1-antitrypsin deficiency, complex disorders such as asthma, allergy, and lung cancer, infections such as respiratory syncytial virus (RSV) and Pseudomonas, aswell as pulmonary arterial hypertension, transplant rejection, andlung injury.Avariety 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. Althoughmuch has been learned through amyriad 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.

Original languageEnglish
Pages (from-to)3-84
Number of pages82
JournalHuman Gene Therapy
Issue number1
Publication statusPublished - 1 Jan 2017
Externally publishedYes



  • Gene therapy
  • Lung
  • Lung disease
  • Non-viral vectors
  • Viral vectors

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Genetics

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