Daniel Wallman, MD

Research Project:
Studying Genetic Variations in Primary Ciliary Dyskinesia in Understudied Populations

Grant Awarded:

Catalyst Award

Research Topics:

gene therapy
modeling

Research Disease:

primary ciliary dyskinesia

(PCD) is a rare, inherited disorder. It affects cilia, tiny hair-like organs that help the body clear mucus. It is caused by variants in more than 50 genes and lacks targeted therapies to treat it. Our goal is to develop a novel platform to determine the disease-causing capacity of high-priority genetic mutations involved in PCD in understudied populations. We will study variants in four genes that are highly prevalent in understudied non-Caucasian populations where PCD is estimated to be more prevalent than currently observed. We will then study RNA differences in lung cells caused by these variants using single-cell RNA sequencing. These findings will provide the foundation needed to make it easier to confirm that a person has PCD, better understand the genetic underpinnings of the disease, and create human models of PCD that lead to therapy.

Page last updated: October 7, 2024

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