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Researchers

Carrie Miceli, PhD

Researcher
Co-Director
Center for Duchenne Muscular Dystrophy, UCLA
609 Charles E. Young Dr. East
1602 Molecular Science Building
Los Angeles, California, United States

Dr. M. Carrie Miceli is Professor and Vice Chair of Microbiology, Immunology and Molecular Genetics at the David Geffen School of Medicine and the College of Life Sciences at UCLA. She earned her Ph.D. in Immunology from Duke University for studies elucidating T cell mechanisms of human kidney allograft rejection. Her research has broad relevance to cell biology and signal transduction. Recently, she has developed an interest in the role of inflammation in Duchenne Muscular Dystrophy.

In 2005, together with Dr. Nelson, Dr. Miceli developed a program designing and implementing cellular assays for high throughput small molecule screening for DMD-drug discovery. She served as a PI/Co-PI on DOD, NIH and CIRM grants aimed at identifying enhancers of DMD exon skipping; resulting in the discovery of compounds that synergizes with anti-sense oligonucleotide mediated DMD exon skipping, award of a provisional patent, licensing negotiations and collaborations with several biotech companies.

In 2007, Drs. M. Carrie Miceli, Melissa Spencer, and Nelson launched the Center for Duchenne Muscular Dystrophy at UCLA to improve education, accelerate research and promote translation into clinic for the most common lethal genetic disease of childhood. Drs Miceli and Nelson were motivated to focus around DMD discovery and drug development upon the diagnosis of their youngest son, Dylan, with Duchenne Muscular Dystrophy in 2004.

 

Representative Publications:

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

Emerging genetic therapies to treat Duchenne muscular dystrophy

DMD genotype correlations from duchenneConnect: Endogenous exon skipping is a factor in prolonged ambulation for individuals with a defined mutation sub-type

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

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