PPMD is excited to announce that Dr. Carrie Miceli from the Center for Duchenne Muscular Dystrophy at University of California – Los Angeles (UCLA) is the recipient of a $100,000 grant to continue her work to immuno-phenotype cells from people living with Duchenne, Becker, and control patients. Understanding the immune profile and how it changes through the course of disease can provide researchers and clinicians better models of disease pathology and progression.

Abby Bronson, PPMD’s Senior Vice President of Research Strategy, explains this important work: “Although the immune response is known in Duchenne, there remains a gap in our knowledge regarding the specific subsets of immune cells that characterize Duchenne and how they relate to disease progression. The work being performed at UCLA by Dr. Miceli and her team aims to uncover the assorted immune cells present in Duchenne by immuno-phenotyping cells from various patient populations. Samples collected from Duchenne, Becker, and control patients at UCLA will undergo immuno-phenotyping through newer mass cytometry technology.”

Dr. Miceli is grateful to PPMD for supporting this research: “By exposing samples to a panel of validated antibodies, we will be able to characterize and classify the different subsets of immune cells present in Duchenne compared to Becker and control patients. Furthermore, investigating samples from patients at various ages and different severities of Duchenne may explain how the immune cell profile changes throughout the course of the disease and identify links between immune infiltrate, blood immune profle and disease severity. Thanks to this grant from PPMD and with  recent advancements in technology, the number of immune cell markers that can be examined has expanded, allowing for a deeper profiling DMD peripheral blood and immune infiltrate than before. We appreciate our ongoing partnership with the PPMD team.”

Bronson believes that, if successful, data from Dr. Miceli’s research could have long-term benefits to Duchenne research and care: “Better interrogation and characterization of the immune cells present in Duchenne has the potential to improve both care and treatment of disease. This work may also discover blood biomarkers that can be used to monitor disease progression and response to therapeutic interventions. We are hopeful that by revealing various immune cells present in dystrophic muscle, this project may expose new targets for therapeutic intervention.”

For years, PPMD has collaborated with Dr. Miceli and the team at the Center for Duchenne Muscular Dystrophy at UCLA – a clinic, which in 2016, was named a Certified Duchenne Care Center by the organization. PPMD’s Certified Duchenne Care Center Program supports standardized, comprehensive care, and services for all people living with Duchenne. To date, 25 clinics have been certified across the U.S., and UCLA was the 12^threcipient of this recognition.

To learn more about PPMD’s robust Research Strategy, click here.