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Human Molecular Genetics|November 26, 2004
Strand bias in oligonucleotide-mediated dystrophin gene editingCarmen Bertoni, Glenn E Morris, Thomas A Rando
Human Molecular Genetics|June 15, 2010
Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNsRefik Kayali, Frederic Bury, McIver Ballard, et al.
Biology|November 19, 2014
High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomicsThomas J J Gintjee, Alvin S H Magh, Carmen Bertoni
Methods in Molecular Biology (Clifton, N.J.)|February 22, 2014
In utero delivery of oligodeoxynucleotides for gene correctionLingzhi Cai, Bhanu Munil Koppanati, Carmen Bertoni, et al.
Human Molecular Genetics|April 25, 2013
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 regenerationMichael V Khanjyan, Jonathan Yang, Refik Kayali, et al.
Human Molecular Genetics|June 14, 2012
Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophyRefik Kayali, Jin-Mo Ku, Gregory Khitrov, et al.
Human Gene Therapy|September 1, 2006
Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA deliveryJoylette L Portlock, Annahita Keravala, Carmen Bertoni, et al.
Human Molecular Genetics|May 18, 2011
Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellumLiutao Du, Refik Kayali, Carmen Bertoni, et al.
Nature Communications|June 28, 2019
Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophySteven S Welc, Ivan Flores, Michelle Wehling-Henricks, et al.
Proceedings of the National Academy of Sciences of the United States of America|January 3, 2006
Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integrationCarmen Bertoni, Sohail Jarrahian, Thurman M Wheeler, et al.
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