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- Principles and general themes
-
1. Oncolytic viruses: strategies, applications and challenges
- Dr. Stephen J. Russell
-
2. Directed evolution of AAV delivery systems for clinical gene therapy
- Prof. David Schaffer
-
6. The host response: adaptive immune response to viral vector delivery
- Prof. Roland W. Herzog
-
7. Gene therapy and virotherapy in the treatment of cancer
- Prof. Leonard Seymour
-
8. Gene therapy for the muscular dystrophies
- Prof. Jeff Chamberlain
- Major gene transfer platforms and gene therapy strategies
-
9. Gammaretroviral vectors: biology, design and applications
- Prof. Axel Schambach
-
13. Surface-mediated targeting of lentiviral vectors
- Prof. Dr. Christian Buchholz
-
14. Gene transfer and gene therapy
- Dr. David A. Williams
-
15. Tracking vector insertion sites to explore the biology of transduced cells in vivo
- Prof. Dr. Christof Von Kalle
-
16. Advances in gene therapy for respiratory diseases 1
- Prof. John F. Engelhardt
-
17. Advances in gene therapy for respiratory diseases 2
- Prof. John F. Engelhardt
-
20. Gene therapy for hemophilia
- Prof. Katherine High
- New technologies for sequence-specific editing of gene expression
-
21. Helper-dependent adenoviral vectors for gene therapy
- Prof. Nicola Brunetti-Pierri
-
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
-
23. RNAi for neurological diseases
- Prof. Beverly L. Davidson
-
24. Directed evolution of novel adeno-associated viral vectors for gene therapy
- Prof. David Schaffer
Printable Handouts
Navigable Slide Index
- Introduction
- Financial disclosures
- Talk goals
- Anatomy of human lung epithelia
- Proximal airway epithelial cell types in the human lung
- Distal conducting airway epithelial cell types
- Inherited and acquired lung diseases
- Stem cell targets for gene editing in the lung
- Gene structure/function 101
- Four general approaches to complement genetic defects by gene therapy
- Interacting preclinical research domains required for developing lung gene therapy
- Cystic Fibrosis (CF): multi-organ disorder
- Disease example: molecular defects in CF
- Animal models of CF
- Species differences in airway cellular anatomy
- Comparative airway cell biology
- Lung and pancreatic phenotype in larger CF animal models
- CFTR expressing epithelial target cell types
- CFTR expressing target cell types: proximal airway
- Gene therapy in a multicellular epithelium requires knowledge of how the target protein functions
- Mechanisms of CFTR function are important
- CFTR gene transfer to a CF airway
- 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:
Therapeutic Areas:
Talk Citation
Engelhardt, J.F. (2023, November 30). Advances in gene therapy for respiratory diseases 1 [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved November 9, 2024, from https://doi.org/10.69645/INAS4440.Export Citation (RIS)
Publication History
Financial Disclosures
- Prof. John F. Engelhardt has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
Advances in gene therapy for respiratory diseases 1
Published on November 30, 2023
22 min
A selection of talks on Genetics & Epigenetics
Transcript
Please wait while the transcript is being prepared...
0:00
Thank you for
joining. My name is
John Engelhardt and
I'm a professor
in the Department of
Anatomy and Cell Biology
at the University of Iowa.
I'm Director of the
Center for Gene Therapy
and the National Ferret
Resource Center.
I'm going to be talking
to you today about
advances in gene therapy
for respiratory diseases.
0:21
These are my financial
disclosures.
0:25
The goals of my talk I'm
going to outline here.
I'm first going to talk about
an introduction to
the cellular anatomy
of the lung and its diverse
cellular targets
for gene therapy.
I'm going to give
some disease examples
of target regions of the
lung for gene therapy.
I'm going to discuss
regional diversity of
epithelial stem cell targets
that are important
for gene editing.
I'm going to next discuss
the importance of
animal models in the testing
of gene therapy approaches.
Then I'll use cystic fibrosis
as an example of
lung disease for
testing gene therapy
and cell biology
considerations that impact
the approaches that are used.
Lastly, I'm going to talk about
general approaches for
vector systems that can
be used for gene
therapy in the lung
in applications of
cystic fibrosis.
1:17
Now the anatomy of the
human lung is very complex.
There are regional differences
in the types of cells
that exist throughout
both the proximal airway,
which includes the
trachea, the bronchi,
and the cartilaginous
regions shown here in white.
These are within Regions 1,
and 2 that also contain
submucosal glands as you
see over here on the right.
Terminal bronchioles
are composed of
a more simple epithelial layer
and respiratory
bronchioles bridge
the gap between the alveoli,
which is the gas exchanging
air space and the
conducting airways.