In the Doerfler lab, we seek to enhance emerging genetic therapies by better understanding the cis-regulatory elements governing gene expression and the mechanisms that maintain genome stability.
More precisely, we’re focused on two key areas: the functional genomics of hemoglobin switching, and how blood stem cells respond to DNA damage. Beta-hemoglobinopathies are a group of inherited blood disorders that affect the production and/or structure of beta-globin, a protein essential to oxygen transport. Given the strong genetic underpinnings of these diseases and their devastating effects, they are excellent candidates for gene therapy interventions. Several such treatments have cleared FDA approval and are now transforming patients’ lives. However, gene therapy remains an expensive and physically taxing intervention. For patients to endure these costs and live with edited hematopoietic stem cells for the rest of their lives, we must continue to seek to understand everything we can about the technology and the biology.
Common techniques you'll find us doing:
- Cell culture
- Gene editing
- PCR: basic, quantitative, and digital
- Single-cell sequencing
- Bacterial culture
- RNAscope
- Western blotting
- ICC/IF
Interested in joining or rotating? Email pdoerfler@versiti.org