Neonatologist and researcher Dr. Sandra Leibel discusses her research into a particular gene therapy process involving a lung organoid model.

She explains her research and surrounding issues, including

• the basics of lung research, and specifically the importance of the surfactant process in keeping lungs from collapsing;
• how mutations lead to the need for surfactant protein b deficiency treatment in babies; and 
• how her model showed positive treatment possibilities but what must happen before treatment is available clinically.

Dr. Sandra Leibel is an assistant clinical professor in pediatrics at UC San Diego School of Medicine and a neonatologist specialist at Rady Children's Hospital in San Diego.  She's currently focused on the lab side of her work involving a gene therapy process.

Dr. Leibel created a model using induced pluripotent stem cells, or embryonic stem cells, and differentiated them into three-dimensional lung organoids. She's using these models to test a possible surfactant protein b deficiency treatment. 

She explains to listeners the basics of lung geography and mechanics and how of the 40 different lung cell types, she uses the epithelial cells in her model. She describes the surfactant production that happens in the distal portion of the lung, which is the furthest portion, yet serves the whole lung by reducing surface tension and keeping our lungs from collapsing. 

These alveolar type 2 cells can undergo a mutation during embryonic development that damages the b protein of those cell; they cannot then produce effective surfactant. These babies are born needing to be on a breathing machine until they are able to get a lung transplant.

However, she's found an exciting advancement in her research, namely that by introducing a virus vector that carried a healthy b gene, she measured signs of the model cells completely normalizing into surfactant-producing cells. In other words, she was able to cure the disease in a dish.  She explains the implications of this, the timing for clinical use, and other related issues.

For more, google her name and see her page at UC San Diego: https://profiles.ucsd.edu/sandra.leibel