Zobrazeno 1 - 10
of 288
pro vyhledávání: '"Jennifer E Morgan"'
Autor:
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P. Kelly, Federica Montanaro, Jennifer E. Morgan, Maaike van Putten, Annemieke Aartsma-Rus, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Publikováno v:
Biomedicines, Vol 11, Iss 12, p 3243 (2023)
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that disrupt the open reading frame and thus prevent production of functional dystrophin proteins. Recent advances in DMD treatment, notably exon skipping and AAV gene therapy,
Externí odkaz:
https://doaj.org/article/29a1434a711345e993444660698a38fe
Autor:
Charlotte Spicer, Ching‐Hua Lu, Francesco Catapano, Mariacristina Scoto, Irina Zaharieva, Andrea Malaspina, Jennifer E. Morgan, Linda Greensmith, Francesco Muntoni, Haiyan Zhou
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 8, Iss 4, Pp 866-876 (2021)
Abstract Objectives To investigate the levels of neurofilaments (NFs) in transgenic mice and patients with spinal muscular atrophy (SMA), and to evaluate their efficacy as a biomarker in SMA. Methods The levels of NF mRNA transcripts were measured by
Externí odkaz:
https://doaj.org/article/a17fa681b1774cb0a695e1e759c2ff6f
Autor:
Amel Saoudi, Claire Fergus, Talia Gileadi, Federica Montanaro, Jennifer E. Morgan, Vincent P. Kelly, Thomas Tensorer, Luis Garcia, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Publikováno v:
Cells, Vol 12, Iss 6, p 908 (2023)
Nucleic acid-based therapies have demonstrated great potential for the treatment of monogenetic diseases, including neurologic disorders. To date, regulatory approval has been received for a dozen antisense oligonucleotides (ASOs); however, these che
Externí odkaz:
https://doaj.org/article/beb2afcd99a94239af7d9e7e2d20f5e8
Autor:
Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, Lucy Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot, Julie Dumonceaux, Jennifer E. Morgan, Paul Gissen, George Dickson, Francesco Muntoni
Publikováno v:
Journal of Cachexia, Sarcopenia and Muscle, Vol 11, Iss 3, Pp 768-782 (2020)
Abstract Background Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1) gene that lead to SMN deficiency. Different SMN‐restoring therapies substantially prolong survival and function in transgenic mice
Externí odkaz:
https://doaj.org/article/833a799c692e44558f9719c738478c3a
Autor:
Jennifer E. Morgan, Alexandre Prola, Virginie Mariot, Veronica Pini, Jinhong Meng, Christophe Hourde, Julie Dumonceaux, Francesco Conti, Frederic Relaix, Francois-Jerôme Authier, Laurent Tiret, Francesco Muntoni, Maximilien Bencze
Publikováno v:
Nature Communications, Vol 9, Iss 1, Pp 1-10 (2018)
Muscular dystrophies are characterised by extensive myofibre cell death. Here Morgan et al. show that RIPK3-mediated necroptosis contributes to myofibre cell death in Duchenne muscular dystrophy, and that RIPK3 deletion protects dystrophic mice again
Externí odkaz:
https://doaj.org/article/e3702b52675e4cdd82568be2bf38a982
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-11 (2017)
Abstract Viral vectors are effective tools in gene therapy, but their limited packaging capacity can be restrictive. Larger clinically-relevant vectors are needed. Foamy viruses have the largest genomes among mammalian retroviruses and their vectors
Externí odkaz:
https://doaj.org/article/a26dc39854a54893898e2bcd915a211e
Autor:
Luisa Boldrin, Jacob A. Ross, Charlotte Whitmore, Bruno Doreste, Charlotte Beaver, Ayad Eddaoudi, Daniel J. Pearce, Jennifer E. Morgan
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-16 (2017)
Abstract Loss of skeletal muscle mass and function occurs with increasing age. Calorie restriction (CR) increases the lifespan of C57Bl/6 mice, but not in the shorter-lived DBA/2 strain. There is some evidence that calorie restriction reduces or dela
Externí odkaz:
https://doaj.org/article/074cae7fc6c94624a824616992ad4d08
Autor:
John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-10 (2017)
Abstract Duchenne Muscular Dystrophy (DMD) is caused by a lack of dystrophin expression in patient muscle fibres. Current DMD gene therapy strategies rely on the expression of internally deleted forms of dystrophin, missing important functional domai
Externí odkaz:
https://doaj.org/article/a4e4bc5af0674a76abc106a806bbacbb
Autor:
Valentina Sardone, Matthew Ellis, Silvia Torelli, Lucy Feng, Darren Chambers, Deborah Eastwood, Caroline Sewry, Rahul Phadke, Jennifer E Morgan, Francesco Muntoni
Publikováno v:
PLoS ONE, Vol 13, Iss 3, p e0194540 (2018)
Clinical trials using strategies aimed at inducing dystrophin expression in Duchenne muscular dystrophy (DMD) are underway or at advanced planning stage, including splice switching antisense oligonucleotides (AON), drugs to induce read-through of non
Externí odkaz:
https://doaj.org/article/cb7c92cc16454711a4912773fe14b1a9
Publikováno v:
Journal of Neuromuscular Diseases. 8:S173-S179
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::517162a152aaac28a1c0a3ef87c77d19
https://doi.org/10.3233/jnd-219010
https://doi.org/10.3233/jnd-219010