California NanoSystems Institute
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Carmen Bertoni, Ph.D.

Associate Professor, Neurology
Member, CTSI, California NanoSystems Institute


Contact Information:
Email Address:
Mailing Address: 710 Westwood Plaza
4145 RNRC
Los Angeles, CA 90095

Selected Publications:

Bertoni Carmen, Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells, Frontiers in physiology, 2014, 5, 148.
Nik-Ahd Farnoosh, Bertoni Carmen, Ex vivo gene editing of the dystrophin gene in muscle stem cells using peptide nucleic acid single stranded oligodeoxynucleotides (PNA-SsODNs) induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophy, Stem cells (Dayton, Ohio), 2014.
Cai Lingzhi, Koppanati Bhanu Munil, Bertoni Carmen, Clemens Paula R, In utero delivery of oligodeoxynucleotides for gene correction, Methods in molecular biology (Clifton, N.J.), 2014, 1114, 399-411.
Bertoni Carmen, Methods for the assessment of ssODN-mediated gene correction frequencies in muscle cells, Methods in molecular biology (Clifton, N.J.), 2014, 1114, 59-83.
Khanjyan Michael V, Yang Jonathan, Kayali Refik, Caldwell Thomas, Bertoni Carmen, A high-content, high-throughput siRNA screen identifies cyclin D2 as a potent regulator of muscle progenitor cell fusion and a target to enhance muscle regeneration, Human molecular genetics, 2013, 22 (16), 3283-95.
Bertoni Carmen, Therapy development for neuromuscular diseases: translating hope into promise, Future neurology, 2013, 8 (4).
Kayali Refik, Ku Jin-Mo, Khitrov Gregory, Jung Michael E, Prikhodko Olga, Bertoni Carmen, Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy, Human molecular genetics, 2012, 21 (18), 4007-20.
Du Liutao, Kayali Refik, Bertoni Carmen, Fike Francesca, Hu Hailiang, Iversen Patrick L, Gatti Richard A, Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum, Human molecular genetics, 2011, 20 (16), 3151-60.
Kayali Refik, Bury Frederic, Ballard McIver, Bertoni Carmen, Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs, Human molecular genetics, 2010, 19 (16), 3266-81.
Bertoni Carmen, Rustagi Arjun, Rando Thomas A, Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides, Nucleic acids research, 2009, 37 (22), 7468-82.
Du Liutao, Damoiseaux Robert, Nahas Shareef, Gao Kun, Hu Hailiang, Pollard Julianne M, Goldstine Jimena, Jung Michael E, Henning Susanne M, Bertoni Carmen, Gatti Richard A, Nonaminoglycoside compounds induce readthrough of nonsense mutations, The Journal of experimental medicine, 2009, 206 (10), 2285-97.
Bertoni Carmen, Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides, Frontiers in bioscience : a journal and virtual library, 2008, 13 (10), 517-27.
Portlock Joylette L, Keravala Annahita, Bertoni Carmen, Lee Solomon, Rando Thomas A, Calos Michele P, Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery, Human gene therapy, 2006, 17 (8), 871-6.
Bertoni Carmen, Jarrahian Sohail, Wheeler Thurman M, Li Yining, Olivares Eric C, Calos Michele P, Rando Thomas A, Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration, Proceedings of the National Academy of Sciences of the United States of America, 2006, 103 (2), 419-24.
Bertoni Carmen, Morris Glenn E, Rando Thomas A, Strand bias in oligonucleotide-mediated dystrophin gene editing, Human molecular genetics, 2005, 14 (2), 221-33.
Bertoni Carmen, Lau Catherine, Rando Thomas A, Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping, Human molecular genetics, 2003, 12 (10), 1087-99.
Bertoni Carmen, Rando Thomas A, Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides, Human gene therapy, 2002, 13 (6), 707-18.