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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Nature Medicine, 2015-03, Vol.21 (3), p.270-275

2015

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Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy

Current pharmaceutical design, 2013, Vol.19 (16), p.2948-2962

2013

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Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

Human molecular genetics, 2008-12, Vol.17 (24), p.3909-3918

2008

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Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

Nucleic acids research, 2008-11, Vol.36 (20), p.6418-6428

2008

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Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

Human molecular genetics, 2015-12, Vol.24 (23), p.6756-6768

2015

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Novel EGFP reporter cell and mouse models for sensitive imaging and quantification of exon skipping

Scientific reports, 2020-06, Vol.10 (1), p.10110-10110, Article 10110

2020

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Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

BioMed research international, 2013-01, Vol.2013 (2013), p.1-8

2013

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Therapeutic approaches for Duchenne muscular dystrophy

Nature reviews. Drug discovery, 2023-11, Vol.22 (11), p.917-934

2023

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Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing

RNA biology, 2021-07, Vol.18 (7), p.1048-1062

2021

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Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

Human molecular genetics, 2013-12, Vol.22 (24), p.4914-4928

2013

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Splicing therapy for neuromuscular disease

Molecular and cellular neuroscience, 2013-09, Vol.56, p.169-185

2013

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Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice

Nature communications, 2016-03, Vol.7 (1), p.10981-11, Article 10981

2016

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A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice

Human molecular genetics, 2009-11, Vol.18 (22), p.4405-4414

2009

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Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Nucleic acids research, 2015-01, Vol.43 (1), p.29-39

2015

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Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

Scientific reports, 2015-11, Vol.5 (1), p.17014-17014, Article 17014

2015

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Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Scientific reports, 2015-03, Vol.5 (1), p.8986-8986, Article 8986

2015

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Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Molecular therapy, 2010-10, Vol.18 (10), p.1822-1829

2010

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Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

Human molecular genetics, 2011-02, Vol.20 (3), p.413-421

2011

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Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

Molecular therapy, 2010-04, Vol.18 (4), p.819-827

2010

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Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy

Scientific reports, 2015-06, Vol.5 (1), p.11632, Article 11632

2015

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Dystrophin involvement in peripheral circadian SRF signalling

Life science alliance, 2021-10, Vol.4 (10), p.e202101014

2021

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In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy

The journal of gene medicine, 2010-04, Vol.12 (4), p.354-364

2010

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Design and application of bispecific splice-switching oligonucleotides

Nucleic acid therapeutics, 2014-02, Vol.24 (1), p.13-24

2014

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Optimizing tissue-specific antisense oligonucleotide-peptide conjugates

Methods in molecular biology (Clifton, N.J.), 2012, Vol.867, p.415

2012

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Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

Novel EGFP reporter cell and mouse models for sensitive imaging and quantification of exon skipping

Development of Multiexon Skipping Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

Therapeutic approaches for Duchenne muscular dystrophy

Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

Splicing therapy for neuromuscular disease

Hexose enhances oligonucleotide delivery and exon skipping in dystrophin-deficient mdx mice

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice

Bi-specific splice-switching PMO oligonucleotides conjugated via a single peptide active in a mouse model of Duchenne muscular dystrophy

Identification of novel, therapy-responsive protein biomarkers in a mouse model of Duchenne muscular dystrophy by aptamer-based serum proteomics

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy

Dystrophin involvement in peripheral circadian SRF signalling

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy

Design and application of bispecific splice-switching oligonucleotides

Optimizing tissue-specific antisense oligonucleotide-peptide conjugates

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