Splice-Switching Therapy for Spinal Muscular Atrophy

por Meijboom, Katharina E., Wood, Matthew J.A., McClorey, Graham
Publicado 2017

Combining multiomics and drug perturbation profiles to identify muscle-specific treatments for spinal muscular atrophy

por Meijboom, Katharina E., Volpato, Viola, Monzón-Sandoval, Jimena, Hoolachan, Joseph M., Hammond, Suzan M., Abendroth, Frank, de Jong, Olivier G., Hazell, Gareth, Ahlskog, Nina, Wood, Matthew J.A., Webber, Caleb, Bowerman, Melissa
Publicado 2021

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

por Walter, Lisa M, Koch, Christiane E, Betts, Corinne A, Ahlskog, Nina, Meijboom, Katharina E, van Westering, Tirsa L E, Hazell, Gareth, Bhomra, Amarjit, Claus, Peter, Oster, Henrik, Wood, Matthew J A, Bowerman, Melissa
Publicado 2018

Dysregulation of Tweak and Fn14 in skeletal muscle of spinal muscular atrophy mice

por Meijboom, Katharina E., Sutton, Emma R., McCallion, Eve, McFall, Emily, Anthony, Daniel, Edwards, Benjamin, Kubinski, Sabrina, Tapken, Ines, Bünermann, Ines, Hazell, Gareth, Ahlskog, Nina, Claus, Peter, Davies, Kay E., Kothary, Rashmi, Wood, Matthew J. A., Bowerman, Melissa
Publicado 2022

Reducing Ventral Tegmental Dopamine D2 Receptor Expression Selectively Boosts Incentive Motivation

por de Jong, Johannes W, Roelofs, Theresia J M, Mol, Frédérique M U, Hillen, Anne E J, Meijboom, Katharina E, Luijendijk, Mieneke C M, van der Eerden, Harrie A M, Garner, Keith M, Vanderschuren, Louk J M J, Adan, Roger A H
Publicado 2015

Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice

por Walter, Lisa M., Deguise, Marc-Olivier, Meijboom, Katharina E., Betts, Corinne A., Ahlskog, Nina, van Westering, Tirsa L.E., Hazell, Gareth, McFall, Emily, Kordala, Anna, Hammond, Suzan M., Abendroth, Frank, Murray, Lyndsay M., Shorrock, Hannah K., Prosdocimo, Domenick A., Haldar, Saptarsi M., Jain, Mukesh K., Gillingwater, Thomas H., Claus, Peter, Kothary, Rashmi, Wood, Matthew J.A., Bowerman, Melissa
Publicado 2018

Dystrophin involvement in peripheral circadian SRF signalling

por Betts, Corinne A, Jagannath, Aarti, van Westering, Tirsa LE, Bowerman, Melissa, Banerjee, Subhashis, Meng, Jinhong, Falzarano, Maria Sofia, Cravo, Lara, McClorey, Graham, Meijboom, Katharina E, Bhomra, Amarjit, Lim, Wooi Fang, Rinaldi, Carlo, Counsell, John R, Chwalenia, Katarzyna, O’Donovan, Elizabeth, Saleh, Amer F, Gait, Michael J, Morgan, Jennifer E, Ferlini, Alessandra, Foster, Russell G, Wood, Matthew JA
Publicado 2021

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro

por Meijboom, Katharina E., Abdallah, Abbas, Fordham, Nicholas P., Nagase, Hiroko, Rodriguez, Tomás, Kraus, Carolyn, Gendron, Tania F., Krishnan, Gopinath, Esanov, Rustam, Andrade, Nadja S., Rybin, Matthew J., Ramic, Melina, Stephens, Zachary D., Edraki, Alireza, Blackwood, Meghan T., Kahriman, Aydan, Henninger, Nils, Kocher, Jean-Pierre A., Benatar, Michael, Brodsky, Michael H., Petrucelli, Leonard, Gao, Fen-Biao, Sontheimer, Erik J., Brown, Robert H., Zeier, Zane, Mueller, Christian
Publicado 2022