Printable Handouts

PDF

Navigable Slide Index

1. Introduction
2. Financial disclosures
3. Talk goals
4. Anatomy of human lung epithelia
5. Proximal airway epithelial cell types in the human lung
6. Distal conducting airway epithelial cell types
7. Inherited and acquired lung diseases
8. Stem cell targets for gene editing in the lung
9. Gene structure/function 101
10. Four general approaches to complement genetic defects by gene therapy
11. Interacting preclinical research domains required for developing lung gene therapy
12. Cystic Fibrosis (CF): multi-organ disorder
13. Disease example: molecular defects in CF
14. Animal models of CF
15. Species differences in airway cellular anatomy
16. Comparative airway cell biology
17. Lung and pancreatic phenotype in larger CF animal models
18. CFTR expressing epithelial target cell types
19. CFTR expressing target cell types: proximal airway
20. Gene therapy in a multicellular epithelium requires knowledge of how the target protein functions
21. Mechanisms of CFTR function are important
22. CFTR gene transfer to a CF airway
23. Thank you for listening

Topics Covered

• Human lung epithelia
• Proximal airway and distal conducting epithelial cell types
• Inherited and acquired lung diseases
• Stem cell targets for gene editing
• Gene transfer and gene editing
• Cystic Fibrosis (CF)
• Animal models of CF
• Comparative airway cell biology
• CFTR gene transfer for CF

Links

Series:

• Gene Transfer and Gene Therapy
• Periodic Reports: Advances in Clinical Interventions and Research Platforms

Categories:

• Genetics & Epigenetics
• Immunology

Therapeutic Areas:

• Respiratory Diseases

Talk Citation

Publication History

• Published on November 30, 2023

Financial Disclosures

• Prof. John F. Engelhardt has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.