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Contact Info:

Email address: cbertoni@ucla.edu

ADDRESS:
Department of Neurology
David Geffen School of Medicine
710 Westwood Plaza
4145 Reed NRC
Los Angeles, CA 90095-1769
Phone/ Fax: (310) 825-6387

 

The goal of the laboratory is to understand the pathogenesis of muscular dystrophies and to develop effective therapies to treat those disorders. The main area of interest is gene therapy for Duchenne muscular dystrophy (DMD), the most prominent of the neuromuscular disorders that affects primarily new born males.  The identification of genes that control muscle regeneration and muscle repair is a key in developing treatments to slow disease progression. Using sate of the art technologies and proteomic analyses, the Bertoni Lab is optimizing new approaches to potentiate the ability of muscle progenitor cells to regenerate skeletal muscles of Duchenne patients. Ultimately however, restoration of dystrophin, the protein missing in DMD is the best option to a safe and permanent treatment to the disorder. The lab is leading the research in the areas of gene correction and drug development using approaches capable of restoring full-length dystrophin expression.  

 

Dr. Carmen Bertoni is an Associate Professor in the Department of Neurology at UCLA. She received her BS from the University of Perugia, Italy and earned her Ph.D. from the same university after completing her graduate program at Tulane as an exchange student. During her postdoctoral fellowship at Stanford she has developed non-viral mediated gene therapies for muscular dystrophies pioneering gene correction strategies using oligonucleotides.