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. 2017 Mar;56(3):283–290. doi: 10.1165/rcmb.2016-0301PS

Table 2.

Potential CRISPR/Cas9 Gene-Editing Approaches in the Lung

  Prenatal Gene Editing Postnatal In Vivo Gene Editing Postnatal Ex Vivo Gene Editing
Target cells Developing progenitor cells In situ tissue-specific stem cells (i.e., basal cells for cystic fibrosis and alveolar type II cells for surfactant protein deficiency) Harvested stem cells
Advantages Higher efficiency Relative technical ease Patient specific
Immune tolerance Avoids in vivo viral exposure
Disease prevention Ability to select for corrected cells before engraftment
Limitations Technical difficulties including specialized equipment Lower efficiency Challenges with reengraftment
Application Genetic surfactant protein deficiency Cystic fibrosis, alpha-1 antitrypsin deficiency Depends on availability of engraftment assay but could include cystic fibrosis