Exon-skipping advances for Duchenne muscular dystrophy von Echevarría, Lucía, Aupy, Philippine, Goyenvalle, Aurélie

Volltext

Partial Restoration of Brain Dystrophin and Behavioral Deficits by Exon Skipping in the Muscular Dystrophy X‐Linked (mdx) Mouse von Zarrouki, Faouzi, Relizani, Karima, Bizot, Flavien, Tensorer, Thomas, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie

Volltext

Emotional behavior and brain anatomy of the mdx52 mouse model of Duchenne muscular dystrophy von Saoudi, Amel, Zarrouki, Faouzi, Sebrié, Catherine, Izabelle, Charlotte, Goyenvalle, Aurélie, Vaillend, Cyrille

Volltext

RNA-targeted drugs for neuromuscular diseases von Ferlini, Alessandra, Goyenvalle, Aurelie, Muntoni, Francesco

Volltext

Impact of the Inhibition of Organic Anion Transporter on Tricyclo-DNA-Mediated Exon Skipping in the mdx Mouse Model von Bizot, Flavien, Tensorer, Thomas, Garcia, Luis, Goyenvalle, Aurélie

Volltext

The bench to bedside journey of tricyclo-DNA antisense oligonucleotides for the treatment of Duchenne muscular dystrophy von Blitek, Mathilde, Phongsavanh, Xaysongkhame, Goyenvalle, Aurélie

Volltext

RNA splicing modulation: Therapeutic progress and perspectives von Saoudi, Amel, Goyenvalle, Aurélie

Volltext

Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders von Bizot, Flavien, Vulin, Adeline, Goyenvalle, Aurélie

Volltext

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping von GOYENVALLE, Aurélie, BABBS, Arran, WRIGHT, Jordan, WILKINS, Vivienne, POWELL, Dave, GARCIA, Luis, DAVIES, Kay E

Volltext

Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model von Relizani, Karima, Griffith, Graziella, Echevarría, Lucía, Zarrouki, Faouzi, Facchinetti, Patricia, Vaillend, Cyrille, Leumann, Christian, Garcia, Luis, Goyenvalle, Aurélie

Volltext

Abnormal Expression of Synaptic and Extrasynaptic GABAA Receptor Subunits in the Dystrophin-Deficient mdx Mouse von Zarrouki, Faouzi, Goutal, Sébastien, Vacca, Ophélie, Garcia, Luis, Tournier, Nicolas, Goyenvalle, Aurélie, Vaillend, Cyrille

Volltext

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides von Goyenvalle, Aurélie, Jimenez-Mallebrera, Cecilia, van Roon, Willeke, Sewing, Sabine, Krieg, Arthur M, Arechavala-Gomeza, Virginia, Andersson, Patrik

Volltext

Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice von Bizot, Flavien, Goossens, Remko, Tensorer, Thomas, Dmitriev, Sergei, Garcia, Luis, Aartsma-Rus, Annemieke, Spitali, Pietro, Goyenvalle, Aurélie

Volltext

In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment von Faiella, Marika, Botti, Giada, Dalpiaz, Alessandro, Gnudi, Lorenzo, Goyenvalle, Aurélie, Pavan, Barbara, Perrone, Daniela, Bovolenta, Matteo, Marchesi, Elena

Volltext

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping von Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E

Volltext

Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice von Benchaouir, Rachid, Meregalli, Mirella, Farini, Andrea, D'Antona, Giuseppe, Belicchi, Marzia, Goyenvalle, Aurélie, Battistelli, Maurizio, Bresolin, Nereo, Bottinelli, Roberto, Garcia, Luis, Torrente, Yvan

Volltext

Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy von Goyenvalle, Aurélie, Wright, Jordan, Babbs, Arran, Wilkins, Vivienne, Garcia, Luis, Davies, Kay E

Volltext

Viral Vector-Mediated Antisense Therapy for Genetic Diseases von Imbert, Marine, Dias-Florencio, Gabriella, Goyenvalle, Aurélie

Volltext

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice von Aupy, Philippine, Zarrouki, Faouzi, Sandro, Quentin, Gastaldi, Cécile, Buclez, Pierre-Olivier, Mamchaoui, Kamel, Garcia, Luis, Vaillend, Cyrille, Goyenvalle, Aurélie

Volltext

uAUG creating variants in the 5’UTR of ENG causing Hereditary Hemorrhagic Telangiectasia von Soukarieh, Omar, Tillet, Emmanuelle, Proust, Carole, Dupont, Charlène, Jaspard-Vinassa, Béatrice, Soubrier, Florent, Goyenvalle, Aurélie, Eyries, Mélanie, Trégouët, David-Alexandre

Volltext