Publications
- Du L., Kayali R., Bertoni C., Fike F., Hu H., Iversen P.L. & Richard A. Gatti (2011) Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum. Hum. Mol. Genet. 19: 3266-81
- Kayali R., Bury F.J., Ballard M. & Bertoni, C. (2010) Site directed gene repair of the dystrophin gene mediated by PNA-ssODNs. Hum. Mol. Genet. 19: 3266-81
- Bertoni C., Rustagi A., Li Y. & Rando T.A. (2009) “Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides”.Nucleic Acids Res., 37: 7468-7482.
- Du L., Damoiseaux R., Nahas S., Pollard J., Goldstine J., Hu H, Jung M., Bertoni C. & Gatti R. (2009) Identification of compounds with the ability of read-through non-sense mutations. J. Ex. Med. 206: 2285-2297.
- Bertoni C. (2008) Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides. Front. In Biosc. 13, 517-527.
- Portlock J.L., Keravala A., Bertoni C., Lee S., Jarrahian S., Rando T.A. & Calos M.P. (2006) Long-Term Increase in mVEGF164 in Mouse Hindlimb Mediated by Phage FC31 Integrase after Non-Viral DNA Delivery. Hum. Gene Ther. 8: 871-6.
- Bertoni C., Jarrahian S., Wheeler T.M., Yining Li, Olivares E.C., Calos M.P., & Rando T.A. (2006) Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration. Proc. Nat.l Acad. Sci. U S A. 103: 419-24.
- Bertoni C. & Rando T.A. (2005) Strand bias in oligonucleotides-mediated dystrophin gene editing. Hum. Mol. Genet. 14:221-33.
- Bertoni C. (2005) Oligonucleotide Mediated Gene Correction for Muscular Dystrophies. Acta Myol. 24, 194-201.
- Bertoni C., Lau C. & Rando T.A. (2003) Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping. Hum. Mol. Genet. 12: 1087-1099.
- Bertoni C. & Rando T.A. (2002) Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA/DNA chimeric oligonucleotides. Hum. Gene Ther. 13: 707-718.
- Bertoni, C., Y.-T., Li & S.-C. Li.(1999) Catabolism of asialo-GM2 in man and mouse. Specificity of human/mouse chimeric GM2 activator proteins J. Biol. Chem. 274: 28612-28618.
- Yuziuk, J.A., Bertoni, C., Beccari, T., Orlacchio, A., Wu, Y.-Y., Li, S.-C. & Li, Y.-T. (1998) Specificity of mouse GM2 activator protein and β-N-acetylhexosaminidase A and B. J. Biol. Chem. 273: 66-72;
- Bertoni, C., Apolloni, M. G., Stirling, J.L., Li, S.-C., Li, Y.-T., Orlacchio & A., Beccari, T. (1997) Structural organization and expression of the gene for the mouse GM2 activator protein, Mamm. Gen. 8: 90-93;
Chapters
- Bertoni C. & Rando T.A. “Oligonucleotide-mediated gene repair for dystrophin gene mutations”, in Duchenne Muscular Dystrophy: Advances in Therapeutics. JS Chamberlain, TA Rando, eds. Marcel Dekker, Inc., New York.