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 |