Search Results - "Muscular Dystrophy"

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice by Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus

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Determination of muscle fatigue index for strength training in patients with Duchenne dystrophy by Adriano Rodrigues Oliveira, Fernanda Ishida Corrêa, Márcia Moreira Valim, Cláudia Santos Oliveira, João Carlos Ferrari Corrêa

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Epidermolysis bullosa: A report of three cases with novel heterozygous deletions in PLEC and homozygous non sense mutations in COL7A1 genes by Sunitha Tella, Shehnaz Sultana, Sujatha Madireddy, Pratibha Nallari, Venkateshwari Ananthapur

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Shorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMD by Ugur Akpulat, Haicui Wang, Kerstin Becker, Adriana Contreras, Terence A. Partridge, James S. Novak, Sebahattin Cirak

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Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery by Ricardo Mondragon-Gonzalez, Rita C. R. Perlingeiro

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DMD/BMD prenatal diagnosis and treatment expectation in a single centre in China for 15 years by Xingjian Zhong, Siying Cui, Lina Liu, Yuxia Yang, Xiangdong Kong

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Concomitant hypo-hyperdontia: A rare entity by Yin-Lin Wang, Hsing-Han Pan, Hsiao-Hua Chang, Guay-Fen Huang

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Non-invasive fetal sex diagnosis in plasma of early weeks pregnants using droplet digital PCR by Elisabetta D'Aversa, Giulia Breveglieri, Patrizia Pellegatti, Giovanni Guerra, Roberto Gambari, Monica Borgatti

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Structural myocardial involvement in adult patients with type 1 myotonic dystrophy by Upinder K. Dhand, Faisal Raja, Kul Aggarwal

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Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides by Suzan M Hammond, Graham McClorey, Joel Z Nordin, Caroline Godfrey, Sofia Stenler, Kim A Lennox, CI Edvard Smith, Ashley M Jacobi, Miguel A Varela, Yi Lee, Mark A Behlke, Matthew J A Wood, Samir E L Andaloussi

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Dental pulp stem cells: Potential significance in regenerative medicine by Todorović Vera, Marković Dejan, Milošević-Jovčić Nadežda, Petakov Marijana, Balint Bela, Čolić Miodrag, Milenković Ana, Čolak Ivana, Jokanović Vukoman, Nikolić Nebojša

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Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes by Afrooz Rashnonejad, Gholamhossein Amini-Chermahini, Noah K. Taylor, Nicolas Wein, Scott Q. Harper

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Metabolite V, an epoxide species is a minor circulating metabolite in humans following a single oral dose of deflazacort by Ronald Kong, Jiyuan Ma, Brian Beers, Diksha Kaushik, E Lin, Elizabeth Goodwin, Joseph Colacino, Francesco Bibbiani

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Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD by Britt Hanson, Sofia Stenler, Nina Ahlskog, Katarzyna Chwalenia, Nenad Svrzikapa, Anna M.L. Coenen-Stass, Marc S. Weinberg, Matthew J.A. Wood, Thomas C. Roberts

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Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene by Masahito Watanabe, Hitomaru Miyamoto, Kazutoshi Okamoto, Kazuaki Nakano, Hitomi Matsunari, Kanako Kazuki, Koki Hasegawa, Ayuko Uchikura, Shuko Takayanagi, Kazuhiro Umeyama, Yosuke Hiramuki, Elisabeth Kemter, Nikolai Klymuik, Mayuko Kurome, Barbara Kessler, Eckhard Wolf, Yasuhiro Kazuki, Hiroshi Nagashima

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CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients by Claudia Provenzano, Marisa Cappella, Rea Valaperta, Rosanna Cardani, Giovanni Meola, Fabio Martelli, Beatrice Cardinali, Germana Falcone

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Intramuscular Evaluation of Chimeric Locked Nucleic Acid/2'<i>O</i>Methyl-Modified Antisense Oligonucleotides for Targeted Exon 23 Skipping in Mdx Mice by Michaella Georgiadou, Melina Christou, Kleitos Sokratous, Jesper Wengel, Kyriaki Michailidou, Kyriacos Kyriacou, Andrie Koutsoulidou, Nikolaos P. Mastroyiannopoulos, Leonidas A. Phylactou

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A Reduction in Selenoprotein S Amplifies the Inflammatory Profile of Fast-Twitch Skeletal Muscle in the mdx Dystrophic Mouse by Craig Robert Wright, Giselle Larissa Allsopp, Alex Bernard Addinsall, Natasha Lee McRae, Sofianos Andrikopoulos, Nicole Stupka

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Cardiac involvement in myotonic dystrophy by Koroush Khalighi, Archana Kodali, Suman B. Thapamagar, Stanley R. Walker

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Therapeutic phosphorodiamidate morpholino oligonucleotides: Physical properties, solution structures, and folding thermodynamics by Farkhad Maksudov, Evgenii Kliuchnikov, Daniel Pierson, M.L. Ujwal, Kenneth A. Marx, Arani Chanda, Valeri Barsegov

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