C. Bertoni

发表

Site-directed gene repair of the dystrophin gene mediated by PNA-ssODNs.

R. Kayali, F. Bury, McIver Ballard, 2010, Human molecular genetics.

Catabolism of Asialo-GM2 in Man and Mouse

Su-Chen Li, Yu-Teh Li, C. Bertoni, 1999, The Journal of Biological Chemistry.

Purloined mechanisms of bacterial immunity can cure muscular dystrophy.

J. Tidball, C. Bertoni, 2014, Cell metabolism.

With regard to glycohemoglobin measurement: are we sure that high-performance liquid chromatography currently works in the clinical routine?

E. Matteucci, C. Bertoni, O. Giampietro, 1998, Acta Diabetologica.

High Throughput Screening in Duchenne Muscular Dystrophy: From Drug Discovery to Functional Genomics

C. Bertoni, T. Gintjee, A. Magh, 2014, Biology.

Genetic and Cell-Mediated Therapies for Duchenne Muscular Dystrophy

C. Bertoni, J. Baglieri, 2015 .

Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.

P. Iversen, R. Gatti, R. Kayali, 2011, Human molecular genetics.

Nonaminoglycoside compounds induce readthrough of nonsense mutations

R. Damoiseaux, R. Gatti, S. Nahas, 2009, The Journal of experimental medicine.

Role of donor/recipient Na+/H+ antiport activity as a nonimmunologic predictor of kidney graft outcome.

F. Mosca, E. Matteucci, C. Cosi, 1997, Transplantation proceedings.

The reliability of urinary albumin assay by immunonephelometry in the clinical practice

E. Matteucci, C. Bertoni, O. Giampietro, 1997, Acta Diabetologica.

Long-term increase in mVEGF164 in mouse hindlimb muscle mediated by phage phiC31 integrase after nonviral DNA delivery.

T. Rando, M. Calos, C. Bertoni, 2006, Human gene therapy.

An early diagnosis of kidney involvement in immunologically-mediated multisystem diseases

P. Migliorini, E. Matteucci, C. Bertoni, 2005, Clinical Rheumatology.

Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy

Steven S. Welc, Ying Wang, J. Tidball, 2019, Nature Communications.

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 Dystrophy

C. Bertoni, F. Nik-Ahd, 2014, Stem cells.

Clinical approaches in the treatment of Duchenne muscular dystrophy (DMD) using oligonucleotides.

C. Bertoni, 2008, Frontiers in bioscience : a journal and virtual library.

Strand bias in oligonucleotide-mediated dystrophin gene editing.

T. Rando, G. Morris, C. Bertoni, 2005, Human molecular genetics.

Dystrophin gene repair in mdx muscle precursor cells in vitro and in vivo mediated by RNA-DNA chimeric oligonucleotides.

T. Rando, C. Bertoni, 2002, Human gene therapy.

Urinary excretion rates of multiple renal indicators after kidney transplantation: clinical significance for early graft outcome.

F. Mosca, E. Matteucci, C. Bertoni, 1998, Renal failure.

Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy.

M. Jung, G. Khitrov, R. Kayali, 2012, Human molecular genetics.

Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy

C. Bertoni, John Hyun Namgoong, 2016, Degenerative neurological and neuromuscular disease.

Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells

C. Bertoni, 2014, Front. Physiol..

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.

Jonathan Yang, R. Kayali, C. Bertoni, 2013, Human molecular genetics.

Enhanced gene repair mediated by methyl-CpG-modified single-stranded oligonucleotides

T. Rando, A. Rustagi, C. Bertoni, 2009, Nucleic acids research.

Restoration of dystrophin expression in mdx muscle cells by chimeraplast-mediated exon skipping.

T. Rando, C. Bertoni, C. Lau, 2003, Human molecular genetics.

Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration.

Thomas A Rando, Michele P Calos, Eric C. Olivares, 2006, Proceedings of the National Academy of Sciences of the United States of America.

Specificity of mouse GM2 activator protein and beta-N-acetylhexosaminidases A and B. Similarities and differences with their human counterparts in the catabolism of GM2.

T. Beccari, A. Orlacchio, Yu-Teh Li, 1998, The Journal of biological chemistry.

Structural organization and expression of the gene for the mouse GM2 activator protein

T. Beccari, A. Orlacchio, J. Stirling, 1997, Mammalian Genome.

Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy

Steven S. Welc, J. Tidball, Ying Wang, 2019, Nature Communications.

In utero delivery of oligodeoxynucleotides for gene correction.

P. Clemens, C. Bertoni, B. M. Koppanati, 2014, Methods in molecular biology.

Therapy development for neuromuscular diseases: translating hope into promise.

C. Bertoni, 2013, Future neurology.