• Principles and general themes
• 48 min
  1. Oncolytic viruses: strategies, applications and challenges

  • Dr. Stephen J. Russell
• 44 min
  2. Directed evolution of AAV delivery systems for clinical gene therapy

  • Prof. David Schaffer
• 47 min
  3. Gene transfer strategies: principles, state-of-the-art and the major barriers that need to be overcome 1

  • Prof. Luigi Naldini
• 30 min
  4. Gene transfer strategies: principles, state-of-the-art and the major barriers that need to be overcome 2

  • Prof. Luigi Naldini
• 36 min
  5. Manufacturing viral gene therapy vectors: general approaches and challenges

  • Prof. John T. Gray
• 41 min
  6. The host response: adaptive immune response to viral vector delivery

  • Prof. Roland W. Herzog
• 46 min
  7. Gene therapy and virotherapy in the treatment of cancer

  • Prof. Leonard Seymour
• 66 min
  8. Gene therapy for the muscular dystrophies

  • Prof. Jeff Chamberlain
• Major gene transfer platforms and gene therapy strategies
• 44 min
  9. Gammaretroviral vectors: biology, design and applications

  • Prof. Axel Schambach
• 58 min
  10. Lentiviral vectors: design, biological properties, milestones and current major applications, hazards 1

  • Prof. Luigi Naldini
• 38 min
  11. Lentiviral vectors: design, biological properties, milestones and current major applications, hazards 2

  • Prof. Luigi Naldini
• 50 min
  12. Gene therapy and gene transfer: vector integration preferences and integration site analysis

  • Prof. Frederic Bushman
• 29 min
  13. Surface-mediated targeting of lentiviral vectors

  • Prof. Dr. Christian Buchholz
• 58 min
  14. Gene transfer and gene therapy

  • Dr. David A. Williams
• 35 min
  15. Tracking vector insertion sites to explore the biology of transduced cells in vivo

  • Prof. Dr. Christof Von Kalle
• 22 min
  16. Advances in gene therapy for respiratory diseases 1

  • Prof. John F. Engelhardt
• 41 min
  17. Advances in gene therapy for respiratory diseases 2

  • Prof. John F. Engelhardt
• 37 min
  18. Hematopoietic stem cell gene therapy for the treatment of metachromatic leukodystrophy

  • Dr. Alessandra Biffi
• 59 min
  19. Turning genomic junk into treasure: genetic engineering with the Sleeping Beauty transposon system

  • Dr. Zoltan Ivics
• 56 min
  20. Gene therapy for hemophilia

  • Prof. Katherine High
• New technologies for sequence-specific editing of gene expression
• 57 min
  21. Helper-dependent adenoviral vectors for gene therapy

  • Prof. Nicola Brunetti-Pierri
• 38 min
  22. HSV vectors: approaches to the treatment of chronic pain

  • Prof. Joseph C. Glorioso
• Archived Lectures *These may not cover the latest advances in the field
• 48 min
  23. RNAi for neurological diseases

  • Prof. Beverly L. Davidson
• 52 min
  24. Directed evolution of novel adeno-associated viral vectors for gene therapy

  • Prof. David Schaffer

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.