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

Nature communications, 2019-06, Vol.10 (1), p.2788-17, Article 2788

2019

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

Human molecular genetics, 2011-08, Vol.20 (16), p.3151-3160

2011

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Nonaminoglycoside compounds induce readthrough of nonsense mutations

The Journal of experimental medicine, 2009-09, Vol.206 (10), p.2285-2297

2009

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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, 2013-08, Vol.22 (16), p.3283-3295

2013

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Enhancement of Plasmid-Mediated Gene Therapy for Muscular Dystrophy by Directed Plasmid Integration

Proceedings of the National Academy of Sciences - PNAS, 2006-01, Vol.103 (2), p.419-424

2006

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Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells

Frontiers in physiology, 2014-04, Vol.5, p.148-148

Bertoni, Carmen

2014

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High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics

Biology, 2014-11, Vol.3 (4), p.752-780

2014

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

Frontiers in bioscience, 2008-01, Vol.13 (13), p.517

Bertoni, Carmen

2008

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Therapy Development For Neuromuscular Diseases: Translating Hope Into Promise

Future neurology, 2013-07, Vol.8 (4), p.399-401

Bertoni, Carmen

2013

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

Human molecular genetics, 2012-09, Vol.21 (18), p.4007-4020

2012

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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

Stem cells (Dayton, Ohio), 2014-07, Vol.32 (7), p.1817-1830

2014

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

Human molecular genetics, 2010-08, Vol.19 (16), p.3266-3281

2010

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Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy

Degenerative Neurological and Neuromuscular Disease, 2016-01, Vol.6, p.37-48

2016

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Purloined Mechanisms of Bacterial Immunity Can Cure Muscular Dystrophy

Cell metabolism, 2014-12, Vol.20 (6), p.927-929

2014

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

Nucleic acids research, 2009-12, Vol.37 (22), p.7468-7482

2009

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Methods for the assessment of ssODN-mediated gene correction frequencies in muscle cells

Methods in molecular biology (Clifton, N.J.), 2014, Vol.1114, p.59

Bertoni, Carmen

2014

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Oligonucleotide-mediated gene editing for neuromuscular disorders

Acta myologica, 2005-12, Vol.24 (3), p.194

Bertoni, C

2005

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Catabolism of Asialo-GM2 in Man and Mouse

The Journal of biological chemistry, 1999-10, Vol.274 (40), p.28612-28618

1999

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Strand bias in oligonucleotide-mediated dystrophin gene editing

Human molecular genetics, 2005-01, Vol.14 (2), p.221-233

2005

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

Human gene therapy, 2002-04, Vol.13 (6), p.707-718

2002

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

Human molecular genetics, 2003-05, Vol.12 (10), p.1087-1099

2003

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573. Enhanced Level of Gene Correction Mediated by Oligonucleotides Containing CpG Modification in the mdx Mouse Model for Duchenne Muscular Dystrophy

Molecular therapy, 2006-05, Vol.13 (S1), p.S221-S221

2006

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262. Plasmid-Mediated Gene Transfer in mdx Mice Using Mini- and Micro-Dystrophin Constructs

Molecular therapy, 2005-05, Vol.11 (S1), p.S103-S103

2005

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

Human gene therapy, 2006-08, Vol.17 (8), p.871

2006

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Long-Term Increase in mVEGF 164 in Mouse Hindlimb Muscle Mediated by Phage ø C31 Integrase After Nonviral DNA Delivery

Human gene therapy, 2006-08, Vol.17 (8), p.871-876

2006

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

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

Nonaminoglycoside compounds induce readthrough of nonsense mutations

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

Enhancement of Plasmid-Mediated Gene Therapy for Muscular Dystrophy by Directed Plasmid Integration

Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells

High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics

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

Therapy Development For Neuromuscular Diseases: Translating Hope Into Promise

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

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

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

Clinical potential of ataluren in the treatment of Duchenne muscular dystrophy

Purloined Mechanisms of Bacterial Immunity Can Cure Muscular Dystrophy

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

Methods for the assessment of ssODN-mediated gene correction frequencies in muscle cells

Oligonucleotide-mediated gene editing for neuromuscular disorders

Catabolism of Asialo-GM2 in Man and Mouse

Strand bias in oligonucleotide-mediated dystrophin gene editing

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

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

573. Enhanced Level of Gene Correction Mediated by Oligonucleotides Containing CpG Modification in the mdx Mouse Model for Duchenne Muscular Dystrophy

262. Plasmid-Mediated Gene Transfer in mdx Mice Using Mini- and Micro-Dystrophin Constructs

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

Long-Term Increase in mVEGF 164 in Mouse Hindlimb Muscle Mediated by Phage ø C31 Integrase After Nonviral DNA Delivery

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