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List of works by Aurélie Goyenvalle

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

scientific article published on 11 June 2013

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy.

scientific article published on 09 February 2011

Correction to: Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders

scientific article published on 04 August 2020

Correction: Impaired Adaptive Response to Mechanical Overloading in Dystrophic Skeletal Muscle

scientific article published on 27 June 2013

Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders

scientific article published on 21 July 2020

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

scientific article

Diaphragm rescue alone prevents heart dysfunction in dystrophic mice

scientific article published on 09 November 2010

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model

scientific article published on 22 June 2017

Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

scientific article

Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy ⬇️

scientific article published on February 21, 2012

Engineering U7snRNA Gene to Reframe Transcripts ⬇️

scientific article published on January 1, 2012

Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.

scientific article

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

scientific article published on 22 April 2019

Exon-skipping advances for Duchenne muscular dystrophy

scientific article published on 01 August 2018

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

scientific article published on 2 February 2015

Gene and splicing therapies for neuromuscular diseases.

scientific article published on June 2015

Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping

scientific article

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

scientific article published on 11 June 2019

Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

scientific article published on 11 September 2012

Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies.

scientific article

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

scientific article published on 08 April 2020

Rescue of a dystrophin-like protein by exon skipping in vivo restores GABAA-receptor clustering in the hippocampus of the mdx mouse.

scientific article

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

scientific article

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

scientific article

Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides.

scientific article published on 15 June 2015

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

scientific article published on June 2012

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

scientific article published on July 2016

The Journal of Gene Medicine European Society of Gene Therapy Young Investigator Award 2005.

scientific article

The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy.

scientific article

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders.

scientific article published on 22 December 2017

Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides

scientific article (publication date: 27 May 2016)

Therapeutic approaches to muscular dystrophies

scientific article published on 01 June 2012

Therapeutic approaches to muscular dystrophy.

scientific article

Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles.

scientific article

Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping

scientific article published on 01 January 2018

Viral Vector-Mediated Antisense Therapy for Genetic Diseases

scientific article

[An opening in Duchenne muscular dystrophy: persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice]

scientific article published on 01 December 2004

[Functional correction and cognitive improvement in dystrophic mice using splice-switching tricyclo-DNA oligomers]

scientific article published on 01 March 2015

[Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice]

scientific article published on 01 January 2008

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