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收藏分享 收藏 Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model
Doisy, Mathilde; Vacca, Ophelie; Fergus, Claire; Gileadi, Talia; Verhaeg, Minou; Saoudi, Amel; Tensorer, Thomas; Garcia, Luis; Kelly, Vincent P.; Montanaro, Federica; Morgan, Jennifer E.; van Putten, Maaike; Aartsma-Rus, Annemieke; Vaillend, Cyrille; Muntoni, Francesco; Goyenvalle, Aurelie 分享 收藏 分享 收藏 Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse ModelsSaoudi, Amel; Fergus, Claire; Gileadi, Talia; Montanaro, Federica; Morgan, Jennifer E. E.; Kelly, Vincent P. P.; Tensorer, Thomas; Garcia, Luis; Vaillend, Cyrille; Muntoni, Francesco; Goyenvalle, Aurelie 分享 收藏 分享 收藏 High-Throughput Digital Image Analysis Reveals Distinct Patterns of Dystrophin Expression in Dystrophinopathy PatientsTorelli, Silvia; Scaglioni, Domenic; Sardone, Valentina; Ellis, Matthew J.; Domingos, Joana; Jones, Adam; Feng, Lucy; Chambers, Darren; Eastwood, Deborah M.; Leturcq, France; Ben Yaou, Rabah; Urtizberea, Andoni; Sabouraud, Pascal; Barnerias, Christine; Stojkovic, Tanya; Ricci, Enzo; Beuvin, Maud; Bonne, Gisele; Sewry, Caroline A.; Willis, Tracey; Kulshrestha, Richa; Tasca, Giorgio; Phadke, Rahul; Morgan, Jennifer E.; Muntoni, Francesco 分享 收藏 Dystrophin involvement in peripheral circadian SRF signallingBetts, Corinne A.; Jagannath, Aarti; Westering, Tirsa L. E. van; 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 J. A. 分享 收藏 The altered expression of neurofilament in mouse models and patients with spinal muscular atrophySpicer, Charlotte; Lu, Ching-Hua; Catapano, Francesco; Scoto, Mariacristina; Zaharieva, Irina; Malaspina, Andrea; Morgan, Jennifer E.; Greensmith, Linda; Muntoni, Francesco; Zhou, Haiyan 分享 收藏 The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophyScaglioni, Dominic; Catapano, Francesco; Ellis, Matthew; Torelli, Silvia; Chambers, Darren; Feng, Lucy; Beck, Matthew; Sewry, Caroline; Monforte, Mauro; Harriman, Shawn; Koenig, Erica; Malhotra, Jyoti; Popplewell, Linda; Guglieri, Michela; Straub, Volker; Mercuri, Eugenio; Servais, Laurent; Phadke, Rahul; Morgan, Jennifer; Muntoni, Francesco 分享 收藏 Novel free-circulating and extracellular vesicle-derived miRNAs dysregulated in Duchenne muscular dystrophyCatapano, Francesco; Scaglioni, Dominic; Maresh, Kate; Ala, Pierpaolo; Domingos, Joana; Selby, Victoria; Ricotti, Valeria; Phillips, Lauren; Servais, Laurent; Seferian, Andreea; de Groot, Imelda; Krom, Yvonne D.; Voit, Thomas; Verschuuren, J. J. G. M.; Niks, E. H.; Straub, Volker; Morgan, Jennifer; Muntoni, Francesco 分享 收藏 rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathyrAAV相关疗法可完全挽救X连锁肌小管肌病的肌核和肌丝功能Ross, Jacob A.; Tasfaout, Hichem; Levy, Yotam; Morgan, Jennifer; Cowling, Belinda S.; Laporte, Jocelyn; Zanoteli, Edmar; Romero, Norma B.; Lowe, Dawn A.; Jungbluth, Heinz; Lawlor, Michael W.; Mack, David L.; Ochala, Julien 分享 收藏 分享 收藏 Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophyFrank, Diane E.; Schnell, Frederick J.; Akana, Cody; El-Husayni, Saleh H.; Desjardins, Cody A.; Morgan, Jennifer; Charleston, Jay S.; Sardone, Valentina; Domingos, Joana; Dickson, George; Straub, Volker; Guglieri, Michela; Mercuri, Eugenio; Servais, Laurent; Muntoni, Francesco 分享 收藏 A high-throughput digital script for multiplexed immunofluorescent analysis and quantification of sarcolemmal and sarcomeric proteins in muscular dystrophiesScaglioni, Dominic; Ellis, Matthew; Catapano, Francesco; Torelli, Silvia; Chambers, Darren; Feng, Lucy; Sewry, Caroline; Morgan, Jennifer; Muntoni, Francesco; Phadke, Rahul 分享 收藏 Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy肌肉生长抑制素抑制联合反义寡核苷酸治疗可改善脊髓性肌萎缩症的预后Zhou, Haiyan; Meng, Jinhong; Malerba, Alberto; Catapano, Francesco; Sintusek, Palittiya; Jarmin, Susan; Feng, Lucy; Ngoc Lu-Nguyen; Sun, Lianwen; Mariot, Virginie; Dumonceaux, Julie; Morgan, Jennifer E.; Gissen, Paul; Dickson, George; Muntoni, Francesco 分享 收藏 分享 收藏 Necroptosis mediates myofibre death in dystrophin-deficient mice (vol 9, 3655, 2018)Morgan, Jennifer E.; Prola, Alexandre; Mariot, Virginie; Pini, Veronica; Meng, Jinhong; Hourde, Christophe; Dumonceaux, Julie; Conti, Francesco; Relaix, Frederic; Authier, Francois-Jerome; Tiret, Laurent; Muntoni, Francesco; Bencze, Maximilien 分享 收藏 Necroptosis mediates myofibre death in dystrophin-deficient miceMorgan, Jennifer E.; Prola, Alexandre; Mariot, Virginie; Pini, Veronica; Meng, Jinhong; Hourde, Christophe; Dumonceaux, Julie; Conti, Francesco; Relaix, Frederic; Authier, Francois-Jerome; Tiret, Laurent; Muntoni, Francesco; Bencze, Maximilien 分享 收藏 Downregulation of miRNA-29,-23 and-21 in urine of Duchenne muscular dystrophy patientsCatapano, Francesco; Domingos, Joana; Perry, Mark; Ricotti, Valeria; Phillips, Lauren; Servais, Laurent; Seferian, Andreea; de Groot, Imelda; Krom, Yvonne D.; Niks, Erik H.; Verschuuren, Jan J. G. M.; Straub, Volker; Voit, Thomas; Morgan, Jennifer; Muntoni, Francesco 分享 收藏 
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