Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

por Goyenvalle, Aurélie, Davies, Kay E
Publicado 2011

Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides

por Goyenvalle, Aurelie, Leumann, Christian, Garcia, Luis
Publicado 2016

Viral Vector-Mediated Antisense Therapy for Genetic Diseases

por Imbert, Marine, Dias-Florencio, Gabriella, Goyenvalle, Aurélie
Publicado 2017

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

por Aupy, Philippine, Echevarría, Lucía, Relizani, Karima, Goyenvalle, Aurélie
Publicado 2017

Emotional behavior and brain anatomy of the mdx52 mouse model of Duchenne muscular dystrophy

por Saoudi, Amel, Zarrouki, Faouzi, Sebrié, Catherine, Izabelle, Charlotte, Goyenvalle, Aurélie, Vaillend, Cyrille
Publicado 2021

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery

por Eckenfelder, Agathe, Tordo, Julie, Babbs, Arran, Davies, Kay E, Goyenvalle, Aurélie, Danos, Olivier
Publicado 2012

Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked (mdx) Mouse

por Zarrouki, Faouzi, Relizani, Karima, Bizot, Flavien, Tensorer, Thomas, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie
Publicado 2022

Abnormal Expression of Synaptic and Extrasynaptic GABA(A) Receptor Subunits in the Dystrophin-Deficient mdx Mouse

por Zarrouki, Faouzi, Goutal, Sébastien, Vacca, Ophélie, Garcia, Luis, Tournier, Nicolas, Goyenvalle, Aurélie, Vaillend, Cyrille
Publicado 2022

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

por Goyenvalle, Aurélie, Babbs, Arran, Wright, Jordan, Wilkins, Vivienne, Powell, Dave, Garcia, Luis, Davies, Kay E.
Publicado 2012

Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment

por Robin, Valérie, Griffith, Graziella, Carter, John-Paul L., Leumann, Christian J., Garcia, Luis, Goyenvalle, Aurélie
Publicado 2017

Common and Rare 5′UTR Variants Altering Upstream Open Reading Frames in Cardiovascular Genomics

por Soukarieh, Omar, Meguerditchian, Caroline, Proust, Carole, Aïssi, Dylan, Eyries, Mélanie, Goyenvalle, Aurélie, Trégouët, David-Alexandre
Publicado 2022

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

por Omairi, Saleh, Hau, Kwan-Leong, Collin-Hooper, Henry, Montanaro, Federica, Goyenvalle, Aurelie, Garcia, Luis, Patel, Ketan
Publicado 2017

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice

por Bizot, Flavien, Goossens, Remko, Tensorer, Thomas, Dmitriev, Sergei, Garcia, Luis, Aartsma-Rus, Annemieke, Spitali, Pietro, Goyenvalle, Aurélie
Publicado 2022

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

por Relizani, Karima, Griffith, Graziella, Echevarría, Lucía, Zarrouki, Faouzi, Facchinetti, Patricia, Vaillend, Cyrille, Leumann, Christian, Garcia, Luis, Goyenvalle, Aurélie
Publicado 2017

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

por Goyenvalle, Aurélie, Jimenez-Mallebrera, Cecilia, van Roon, Willeke, Sewing, Sabine, Krieg, Arthur M., Arechavala-Gomeza, Virginia, Andersson, Patrik
Publicado 2023

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

por Aupy, Philippine, Zarrouki, Faouzi, Sandro, Quentin, Gastaldi, Cécile, Buclez, Pierre-Olivier, Mamchaoui, Kamel, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie
Publicado 2020

uAUG creating variants in the 5’UTR of ENG causing Hereditary Hemorrhagic Telangiectasia

por Soukarieh, Omar, Tillet, Emmanuelle, Proust, Carole, Dupont, Charlène, Jaspard-Vinassa, Béatrice, Soubrier, Florent, Goyenvalle, Aurélie, Eyries, Mélanie, Trégouët, David-Alexandre
Publicado 2023

Exon Skipping in a Dysf-Missense Mutant Mouse Model

por Malcher, Jakub, Heidt, Leonie, Goyenvalle, Aurélie, Escobar, Helena, Marg, Andreas, Beley, Cyriaque, Benchaouir, Rachid, Bader, Michael, Spuler, Simone, García, Luis, Schöwel, Verena
Publicado 2018

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

por Aupy, Philippine, Echevarría, Lucía, Relizani, Karima, Zarrouki, Faouzi, Haeberli, Adrian, Komisarski, Marek, Tensorer, Thomas, Jouvion, Grégory, Svinartchouk, Fedor, Garcia, Luis, Goyenvalle, Aurélie
Publicado 2019

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

por Bizot, Flavien, Fayssoil, Abdallah, Gastaldi, Cécile, Irawan, Tabitha, Phongsavanh, Xaysongkhame, Mansart, Arnaud, Tensorer, Thomas, Brisebard, Elise, Garcia, Luis, Juliano, Rudolph L, Goyenvalle, Aurélie
Publicado 2023