Zobrazeno 1 - 10
of 59
pro vyhledávání: '"Jinhong Meng"'
Autor:
Jinhong Meng, Marc Moore, John Counsell, Francesco Muntoni, Linda Popplewell, Jennifer Morgan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 491-507 (2022)
Duchenne muscular dystrophy (DMD) is a muscle wasting disorder caused by mutations in the DMD gene. Restoration of full-length dystrophin protein in skeletal muscle would have therapeutic benefit, but lentivirally mediated delivery of such a large ge
Externí odkaz:
https://doaj.org/article/19682bbce5954b47a33dacda7ecb08ec
Autor:
Haiyan Zhou, Ying Hong, Mariacristina Scoto, Alison Thomson, Emma Pead, Tom MacGillivray, Elena Hernandez-Gerez, Francesco Catapano, Jinhong Meng, Qiang Zhang, Gillian Hunter, Hannah K. Shorrock, Thomas K. Ng, Abedallah Hamida, Mathilde Sanson, Giovanni Baranello, Kevin Howell, Thomas H. Gillingwater, Paul Brogan, Dorothy A. Thompson, Simon H. Parson, Francesco Muntoni
Publikováno v:
The Journal of Clinical Investigation, Vol 132, Iss 21 (2022)
Spinal muscular atrophy (SMA) is a neuromuscular disorder due to degeneration of spinal cord motor neurons caused by deficiency of the ubiquitously expressed SMN protein. Here, we present a retinal vascular defect in patients, recapitulated in SMA tr
Externí odkaz:
https://doaj.org/article/7bf2c0654d0b499f9112525f28926aef
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:
Stem Cell Research, Vol 30, Iss , Pp 43-52 (2018)
Cell-mediated gene therapy is a possible means to treat muscular dystrophies like Duchenne muscular dystrophy. Autologous patient stem cells can be genetically-corrected and transplanted back into the patient, without causing immunorejection problems
Externí odkaz:
https://doaj.org/article/e250c819134d460a9a831659def65413
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:
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
Publikováno v:
International Journal of Molecular Sciences, Vol 21, Iss 19, p 7168 (2020)
Background: We are developing a novel therapy for Duchenne muscular dystrophy (DMD), involving the transplantation of autologous, skeletal muscle-derived stem cells that have been genetically corrected to express dystrophin. Dystrophin is normally ex
Externí odkaz:
https://doaj.org/article/06f26ed9600347c0868d8b4552194f16
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 1-1 (2018)
The original version of this article contained an error in Fig. 3. In panel c, the labels ‘mdx’ and ‘mdx Ripk3-/-‘ were inadvertently inverted. This has now been corrected in the PDF and HTML versions of the Article.
Externí odkaz:
https://doaj.org/article/bc571b2b1d674f85a50ecedc8287b7c3
Publikováno v:
PLoS ONE, Vol 6, Iss 3, p e17454 (2011)
BackgroundStem cell transplantation is a promising potential therapy for muscular dystrophies, but for this purpose, the cells need to be systemically-deliverable, give rise to many muscle fibres and functionally reconstitute the satellite cell niche
Externí odkaz:
https://doaj.org/article/fbcc0f9fe4df4cc6bd5bf00df5d41161