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Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles

Molecular therapy, 2017-07, Vol.25 (7), p.1580-1587

2017

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Therapeutic gene silencing strategies for polyglutamine disorders

Trends in genetics, 2010-01, Vol.26 (1), p.29-38

2010

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Genetic therapies for RNA mis-splicing diseases

Trends in genetics, 2011-05, Vol.27 (5), p.196-205

2011

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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

Volltextzugriff (PDF)

Use of cell-penetrating-peptides in oligonucleotide splice switching therapy

Current gene therapy, 2012-06, Vol.12 (3), p.161

2012

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Selective release of muscle-specific, extracellular microRNAs during myogenic differentiation

Human molecular genetics, 2016-09, Vol.25 (18), p.3960-3974

2016

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Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies

Human molecular genetics, 2015-09, Vol.24 (17), p.4916-4932

2015

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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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Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics

Human molecular genetics, 2009-04, Vol.18 (R1), p.R27-R39

2009

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Splice-Switching Therapy for Spinal Muscular Atrophy

Genes, 2017-06, Vol.8 (6), p.161

2017

Volltextzugriff (PDF)

mRNA and microRNA transcriptomics analyses in a murine model of dystrophin loss and therapeutic restoration

Genomics data, 2016-03, Vol.7, p.88-89

2016

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Ribozyme-mediated trans -splicing of a trinucleotide repeat

Nature genetics, 1998-04, Vol.18 (4), p.378-381

1998

Volltextzugriff (PDF)

Therapeutic Potential of Multipotent Mesenchymal Stromal Cells and Their Extracellular Vesicles

Human gene therapy, 2015-08, Vol.26 (8), p.506

2015

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Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Human molecular genetics, 2017-11, Vol.26 (22), p.4441-4450

2017

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Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1

Molecular therapy, 2020-12, Vol.28 (12), p.2527-2539

2020

Volltextzugriff (PDF)

A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models

Human molecular genetics, 2024-02, Vol.33 (5), p.400-425

2024

Volltextzugriff (PDF)

Exosomes and the emerging field of exosome-based gene therapy

Current gene therapy, 2012-08, Vol.12 (4), p.262

2012

Volltextzugriff (PDF)

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

Molecular therapy, 2011-07, Vol.19 (7), p.1295-1303

2011

Volltextzugriff (PDF)

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

Molecular therapy, 2020-10, Vol.28 (10), p.2297-2298

2020

Volltextzugriff (PDF)

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

Volltextzugriff (PDF)

Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts

European journal of human genetics : EJHG, 2014-12, Vol.22 (12), p.1369-1375

2014

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Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Gene therapy, 2019-11, Vol.27 (10-11), p.505-515

2019

Volltextzugriff (PDF)

Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice

Human molecular genetics, 2018-10, Vol.27 (20), p.3582-3597

2018

Volltextzugriff (PDF)

To Skip or Not to Skip: That Is the Question for Duchenne Muscular Dystrophy

Molecular therapy, 2013-12, Vol.21 (12), p.2131-2132

Wood, Matthew J A

2013

Volltextzugriff (PDF)

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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Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles

Therapeutic gene silencing strategies for polyglutamine disorders

Genetic therapies for RNA mis-splicing diseases

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

Use of cell-penetrating-peptides in oligonucleotide splice switching therapy

Selective release of muscle-specific, extracellular microRNAs during myogenic differentiation

Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies

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

Short non-coding RNA biology and neurodegenerative disorders: novel disease targets and therapeutics

Splice-Switching Therapy for Spinal Muscular Atrophy

mRNA and microRNA transcriptomics analyses in a murine model of dystrophin loss and therapeutic restoration

Ribozyme-mediated trans -splicing of a trinucleotide repeat

Therapeutic Potential of Multipotent Mesenchymal Stromal Cells and Their Extracellular Vesicles

Alpha-synuclein induces the unfolded protein response in Parkinson's disease SNCA triplication iPSC-derived neurons

Application of CRISPR-Cas9-Mediated Genome Editing for the Treatment of Myotonic Dystrophy Type 1

A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models

Exosomes and the emerging field of exosome-based gene therapy

Pip5 Transduction Peptides Direct High Efficiency Oligonucleotide-mediated Dystrophin Exon Skipping in Heart and Phenotypic Correction in mdx Mice

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

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

Allele-specific silencing of mutant Ataxin-7 in SCA7 patient-derived fibroblasts

Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice

To Skip or Not to Skip: That Is the Question for Duchenne Muscular Dystrophy

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

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