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Frontiers in Physiology|May 6, 2014
Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cellsCarmen BertoniFrontiers in Bioscience : a Journal and Virtual Library|November 6, 2007
Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotidesCarmen BertoniFuture Neurology|November 26, 2013
Therapy development for neuromuscular diseases: translating hope into promiseCarmen BertoniMethods in Molecular Biology (Clifton, N.J.)|February 22, 2014
Methods for the assessment of ssODN-mediated gene correction frequencies in muscle cellsCarmen BertoniCell Metabolism|December 4, 2014
Purloined mechanisms of bacterial immunity can cure muscular dystrophyJames G Tidball, Carmen BertoniStem Cells (Dayton, Ohio)|April 23, 2014
Ex vivo gene editing of the dystrophin gene in muscle stem cells mediated by peptide nucleic acid single stranded oligodeoxynucleotides induces stable expression of dystrophin in a mouse model for Duchenne muscular dystrophyFarnoosh Nik-Ahd, Carmen BertoniHuman Gene Therapy|April 9, 2002
Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotidesCarmen Bertoni, Thomas A RandoDegenerative Neurological and Neuromuscular Disease|July 28, 2018
Clinical potential of ataluren in the treatment of Duchenne muscular dystrophyJohn Hyun Namgoong, Carmen BertoniHuman Molecular Genetics|April 30, 2003
Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skippingCarmen Bertoni, Catherine Lau, Thomas A RandoNucleic Acids Research|October 27, 2009
Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotidesCarmen Bertoni, Arjun Rustagi, Thomas A RandoPageof 3