Results 41 to 50 of about 55,853 (286)

"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic [PDF]

, 2018
A new line of dystrophic mdx mice on the DBA/2J (D2) background has emerged as a candidate to study the efficacy of therapeutic approaches for Duchenne muscular dystrophy (DMD). These mice harbor genetic polymorphisms that appear to increase the severity
Aartsma-Rus, A., Bogdanik, L, Davies, K, De Luca, A, Demonbruen, Ar, Duan, D, Elsey, D, Fukada, Si, Girgenrath, M, Gonzalez, Jp, Gordish-Dressman, H, Grounds, Md, Heydemann, A, Kreibich, A, Lutz, C, Nagaraju, K, Nichols, A, Partridge, T, Passini, M, Pazze, Ld, Sanarica, F, Schnell, Fj, Spencer, M, Spurney, C, van Putten, M, Wells, Dj, Willmann, R, Yokota, T, Young, Cs, Zhong, Z   +29 more
core   +3 more sources

Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. [PDF]

, 2019
Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models.
Barthélémy, Florian, Douine, Emilie D, Hsu, Christopher, Marcantonio, Eugene E, Miceli, M Carrie, Nelson, Stanley F, Wang, Richard T   +6 more
core   +1 more source

Muscle inactivation of mTOR causes metabolic and dystrophin defects leading to severe myopathy [PDF]

, 2009
Mammalian target of rapamycin (mTOR) is a key regulator of cell growth that associates with raptor and rictor to form the mTOR complex 1 (mTORC1) and mTORC2, respectively.
Baas, Dominique, Bauché, Stéphanie, Bigard, Xavier, Conjard-Duplany, Agnès, Corneloup, Claudine, Defour, Aurélia, Ferrier, Bernard, Ferry, Arnaud, Freyssenet, Damien, Gangloff, Yann-Gaël, Hantaï, Daniel, Koulmann, Nathalie, Kozma, Sara C., Le-Marchand-Brustel, Yannick, Mazelin, Laetitia, Moncollin, Vincent, Mueller, Matthias, Pende, Mario, Richard-Bulteau, Hélène, Risson, Valérie, Roceri, Mila, Romanino, Klaas, Rüegg, Markus A., Sanchez, Hervé, Schaeffer, Laurent, Tanti, Jean-François, Thomas, George, Vignaud, Alban   +27 more
core   +5 more sources

Patient‐Derived 3D Bioprinted Cardiac Organoid Constructs Reveal Key Pathological Features of Duchenne Muscular Dystrophy

Advanced Healthcare Materials, EarlyView.
Patient‐derived cardiac organoids reveal key features of Duchenne muscular dystrophy cardiomyopathy, including apoptosis, oxidative stress, calcium handling defects, and mechanical remodeling. By integrating organoids into alginate–gelatin bioprinted constructs, disease phenotypes are organized into scalable 3D cardiac tissues displaying extracellular ...
Vittoria Marini, Margalida Campaner Socias, Andreas Dimopoulos, Lorenza Rinvenuto, Enrico Pozzo, Diana Stalkopf, Angelo Serafini, Sara Morri, Ashley Wang, Rita La Rovere, Yoke Chin Chai, Sveva Bollini, Geert Bultynck, H. Llewelyn Roderick, Ioannis Papantoniou, Maurilio Sampaolesi   +15 more
wiley   +1 more source

Abnormality of water homeostasis of Muscular dystrophic chicken [PDF]

, 2014
The muscular dystrophy chicken has been studying as model animal of muscular dystrophy for more than 50 years. Recently, the mutation of WW domain containing E3 ubiquitin protein ligase 1 (WWP1) gene has been identified as a responsible for muscular ...
Saito, Noboru
core  

Melt Electrowriting High Resolution Poly(ethylene‐co‐vinyl acetate) Scaffolds for Soft Tissue Engineering

Advanced Healthcare Materials, EarlyView.
This work pioneers melt electrowriting (MEW) of polyethylene vinyl acetate (PEVA) to fabricate ultra‐compliant, high‐resolution scaffolds. By integrating microscale precision with soft tissue‐like biomechanics, PEVA overcomes stiffness‐driven limitations of conventional MEW polymers, establishing a mechanically biomimetic platform for soft tissue ...
Finn Snow, Darcy De Rauch, Lilith Mabel Caballero Aguilar, Darcy Millett, Jasley Wilding McBride, David R Nisbet, Magdalena Kita, Elena Pirogova, Robert Michail Ivan Kapsa, Anita Quigley   +9 more
wiley   +1 more source

Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy.

PLoS ONE, 2014
A few animal models of Duchenne muscular dystrophy (DMD) are available, large ones such as pigs or dogs being expensive and difficult to handle. Mdx (X-linked muscular dystrophy) mice only partially mimic the human disease, with limited chronic muscular ...
Thibaut Larcher, Aude Lafoux, Laurent Tesson, Séverine Remy, Virginie Thepenier, Virginie François, Caroline Le Guiner, Helicia Goubin, Maéva Dutilleul, Lydie Guigand, Gilles Toumaniantz, Anne De Cian, Charlotte Boix, Jean-Baptiste Renaud, Yan Cherel, Carine Giovannangeli, Jean-Paul Concordet, Ignacio Anegon, Corinne Huchet   +18 more
doaj   +1 more source

Model Organisms in the Fight against Muscular Dystrophy: Lessons from Drosophila and Zebrafish

Molecules, 2015
Muscular dystrophies (MD) are a heterogeneous group of genetic disorders that cause muscle weakness, abnormal contractions and muscle wasting, often leading to premature death.
Emilie Plantié, Marta Migocka-Patrzałek, Małgorzata Daczewska, Krzysztof Jagla   +3 more
doaj   +1 more source

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy [PDF]

, 2018
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational “hotspot” in the human DMD gene.
Amoasii, L, Bassel-Duby, R, Caballero, D, Harron, R, Hildyard, J C W, Li, H, Massey, C A, Mireault, A, Olson, E N, Piercy, R J, Sanchez-Ortiz, E, Shelton, J M, Stathopoulou, T-R   +12 more
core   +2 more sources

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source