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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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Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

EMBO molecular medicine, 2021-04, Vol.13 (4), p.e13243-n/a

2021

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Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides

Nucleic acids research, 2022-01, Vol.50 (1), p.17-34

2022

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Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

Science (American Association for the Advancement of Science), 2004-12, Vol.306 (5702), p.1796-1799

2004

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Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping

Methods in molecular biology (Clifton, N.J.), 2018-01, Vol.1828, p.381

2018

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Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Molecular therapy, 2012-11, Vol.20 (11), p.2120-2133

2012

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Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease

Nucleic acid therapeutics, 2019-10, Vol.29 (5), p.256-265

2019

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Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B

PLoS genetics, 2020-04, Vol.16 (4), p.e1008690

2020

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Altered visual processing in the mdx52 mouse model of Duchenne muscular dystrophy

Neurobiology of disease, 2021-05, Vol.152, p.105288-105288, Article 105288

2021

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Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice

Molecular therapy. Nucleic acids, 2023-06, Vol.32, p.173-188

2023

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Exon-skipping advances for Duchenne muscular dystrophy

Human molecular genetics, 2018-08, Vol.27 (R2), p.R163-R172

2018

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Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle

Proceedings of the National Academy of Sciences - PNAS, 2023-01, Vol.120 (2), p.e2206324120-e2206324120

2023

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Therapeutic approaches to muscular dystrophy

Human molecular genetics, 2011-04, Vol.20 (R1), p.R69-R78

2011

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Delivery is key: lessons learnt from developing splice‐switching antisense therapies

EMBO molecular medicine, 2017-05, Vol.9 (5), p.545-557

2017

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Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model

Nucleic acid therapeutics, 2019-06, Vol.29 (3), p.148-160

2019

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Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B

PLoS genetics, 2021-01, Vol.17 (1), p.e1009345-e1009345

2021

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AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy

Molecular therapy, 2013-08, Vol.21 (8), p.1551-1558

2013

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Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Cells (Basel, Switzerland), 2023-03, Vol.12 (6), p.908

2023

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Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

Cells (Basel, Switzerland), 2023-02, Vol.12 (5), p.702

2023

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Splicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophies

Current gene therapy, 2012-06, Vol.12 (3), p.179-191

2012

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Exon Skipping in a Dysf-Missense Mutant Mouse Model

Molecular therapy. Nucleic acids, 2018-12, Vol.13, p.198-207

2018

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Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy

Molecular therapy. Nucleic acids, 2020-03, Vol.19, p.371-383

2020

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Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model

Biomedicines, 2023-12, Vol.11 (12), p.3243

2023

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Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice

Methods in molecular biology (Clifton, N.J.), 2022, Vol.2434, p.371

2022

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

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

Palmitic acid conjugation enhances potency of tricyclo-DNA splice switching oligonucleotides

Rescue of Dystrophic Muscle through U7 snRNA-Mediated Exon Skipping

Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease

Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B

Altered visual processing in the mdx52 mouse model of Duchenne muscular dystrophy

Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice

Exon-skipping advances for Duchenne muscular dystrophy

Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle

Therapeutic approaches to muscular dystrophy

Stakeholder cooperation to overcome challenges in orphan medicine development: the example of Duchenne muscular dystrophy

Delivery is key: lessons learnt from developing splice‐switching antisense therapies

Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model

Correction: Pathological mechanism and antisense oligonucleotide-mediated rescue of a non-coding variant suppressing factor 9 RNA biogenesis leading to hemophilia B

AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Oligonucleotide Enhancing Compound Increases Tricyclo-DNA Mediated Exon-Skipping Efficacy in the Mdx Mouse Model

Splicing modulation mediated by small nuclear RNAs as therapeutic approaches for muscular dystrophies

Exon Skipping in a Dysf-Missense Mutant Mouse Model

Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy

Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model

Preclinical Evaluation of the Renal Toxicity of Oligonucleotide Therapeutics in Mice

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