Work Email Address:
Office Phone Number:
276B Biomedical Sciences Research Building
CAMPUS - 157005
257 Basic Sciences Research Building
Los Angeles, CA 90095
Department of Microbiology, Immunology, and Molecular Genetics
Los Angeles, CA 90095
|Co-Director, Center for Duchenne Muscular Dystrophy|
|Professor, Microbiology, Immunology & Molecular Genetics|
|Member, Cell & Developmental Biology GPB Home Area, Immunity, Microbes & Molecular Pathogenesis GPB Home Area, JCCC Signal Transduction and Therapeutics Program Area|
Dr. M. Carrie Miceli graduated with a BA in Biochemistry and Cell Biology from UCSD. She earned her Ph.D. in Immunology from Duke University for studies elucidating T cell mechanisms of human kidney allograft rejection. Her postdoctoral work at Stanford focused on the molecular basis of T cell antigen recognition and signaling, characterizing roles for CD8, CD4 and Lck in T cell receptor signaling. Since her faculty appointment at UCLA in 1993, her lab has investigated the cellular and molecular basis of T cell immunity. In addition to dissecting molecular mechanisms of T cell activation, tolerance and fate, her research has had broad relevance to cell biology and signal transduction. Recently, she has developed an interest in the role of inflammation in Duchenne Muscular Dystrophy with Dr. Spencer. 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. She directs the High Throughput Screening and Muscular Dystrophy Cell Models Repository Core, which expands banks, distributes and reprograms DMD patient fibroblasts for use in drug development, and serves on the Administrative Core for the NIH funded P30 Muscular Dystrophy Core Center. Dr. Miceli is a Scientific Advisor for PPMD, CureDuchenne, Imaging DMD and the Lily DMD Tadalifil Phase II Clinical Trial and is on the Data and Safety Monitoring Board for the BMD/sIBM Follistatin Gene Transfer Clinical Trial. She reviews grants for PPMD, Dutch-PPMD, NIH and DOD. She has chaired the congressionally mandated DOD DMD study section and both annual PPMD and Duchenne Parent Project Italy Scientific Sessions. She serves as a Vice Chair of Microbiology, Immunology and Molecular Genetics.
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. This multidisciplinary effort has transformed research approaches on campus, providing essential seed grants and an intellectual environment to facilitate progress. The CDMD has organized the first multidiscliplinary care clinic for Duchenne in Southern California, documented to extend lifespan by 10 years and dramatically improve quality of life. The Center is now participating in multiple clinical trials for Duchenne, both serving as a site for CDMD investigator initiated trials as well as participating in multi-center clinical trials sponsored by industry and other academic institutions. Since the first funding of the CDMD Core Center in 2009, the Center has successfully expanded the number of multidisciplinary collaborations focused on muscular dystrophy research and accelerated the discovery and testing of potential therapeutics, with 13 laboratories now focused on Duchenne. At the center of the research accomplishments is the collaborative efforts of Drs. Miceli, Nelson and Spencer’s laboratories to identify enhancers of exon skipping to restore reading frame and a partially functional dystrophin protein. This work was published in Science Translational Medicine in December 2012, featured on the cover with commentary in Science, and was selected by the Director of NIAMS as a research highlight. With funding from the California Institute of Regenerative Medicine, they are now moving combination therapy for exon skipping toward clinical development repair mutant mRNA in situ. This collaborative research endeavor exemplifies the CDMD mission of using basic discovery to identify therapeutic targets, screen for drugs and develop them for human clinical trial in DMD. There are a number of therapeutic strategies poised to translate from the bench into clinic for Duchenne, and the CDMD is committed to facilitating their development. This creates exciting research opportunities on campus, in partnership with biotechnology firms, and in collaboration with extramural researchers. The CDMD facilitates activities on campus through an interdepartmental and inter-institutional approach to bring researchers together through a pilot and feasibility program, bi-weekly seminar series, establishment of a new PhD training program, establishment of cores to facilitate study of muscular dystrophy.
Drs Miceli and Nelson were motivated to re-focus their laboratories around DMD discovery and drug development upon the diagnosis of their youngest son with Duchenne Muscular Dystrophy in 2004.