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Advances in gene therapy for respiratory diseases 1

Part 1 of 2
Published on November 30, 2023   22 min

A selection of talks on Genetics & Epigenetics

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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.

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