HACKENSACK, N.J., May 14, 2019 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced 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 muscular dystrophy (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.
Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the dystrophin gene. Instability of the muscle cell membrane in Duchenne, caused by the absence of dystrophin, leads to an influx of immune and inflammatory cells within the muscle, contributing to disease progression. Becker muscular dystrophy, which is less severe than Duchenne, occurs when dystrophin is manufactured, but not in the normal form or amount.
"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," explains Abby Bronson, PPMD's Senior Vice President of Research Strategy. "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 important 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 profile 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 12th recipient of this recognition.
To learn more about PPMD's robust Research Strategy, click here.
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.
We invest deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite and educate the global Duchenne community.
Everything we do—and everything we have done since our founding in 1994—helps people with Duchenne live longer, stronger lives. We will not rest until every person has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne. Follow PPMD on Facebook, Twitter, and YouTube.
SOURCE Parent Project Muscular Dystrophy