Zoekresultaten - "Exon skipping"

Natural Exon Skipping Sets the Stage for Exon Skipping as Therapy for Dystrophic Epidermolysis Bullosa door Jeroen Bremer, Elisabeth H. van der Heijden, Daryll S. Eichhorn, Rowdy Meijer, Henny H. Lemmink, Hans Scheffer, Richard J. Sinke, Marcel F. Jonkman, Anna M.G. Pasmooij, Peter C. Van den Akker

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Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy door van Ommen Gert Jan B, den Dunnen Johan T, de Gorter David JJ, Terlouw Samuel, van Heiningen Sandra H, Hoogaars Willem MH, Kemaladewi Dwi U, Aartsma-Rus Annemieke, ten Dijke Peter, 't Hoen Peter AC

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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy door van Ommen Gert-Jan B, Janson Anneke AM, Aartsma-Rus Annemieke, van Deutekom Judith CT

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Branchpoints as potential targets of exon-skipping therapies for genetic disorders door Hiroaki Ohara, Motoyasu Hosokawa, Tomonari Awaya, Atsuko Hagiwara, Ryo Kurosawa, Yukiya Sako, Megumu Ogawa, Masashi Ogasawara, Satoru Noguchi, Yuichi Goto, Ryosuke Takahashi, Ichizo Nishino, Masatoshi Hagiwara

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Targeted Exon Skipping to Correct Exon Duplications in the Dystrophin Gene door Kane L Greer, Hanns Lochmüller, Kevin Flanigan, Susan Fletcher, Steve D Wilton

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Exon Skipping in a Dysf-Missense Mutant Mouse Model door Jakub Malcher, Leonie Heidt, Aurélie Goyenvalle, Helena Escobar, Andreas Marg, Cyriaque Beley, Rachid Benchaouir, Michael Bader, Simone Spuler, Luis García, Verena Schöwel

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Targeted Exon Skipping to Address "Leaky" Mutations in the Dystrophin Gene door Sue Fletcher, Carl F. Adkin, Penny Meloni, Brenda Wong, Francesco Muntoni, Ryszard Kole, Clayton Fragall, Kane Greer, Russell Johnsen, Steve D. Wilton

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Identification of exon skipping events associated with Alzheimer's disease in the human hippocampus door Seonggyun Han, Jason E. Miller, Seyoun Byun, Dokyoon Kim, Shannon L. Risacher, Andrew J. Saykin, Younghee Lee, Kwangsik Nho, for Alzheimer's Disease Neuroimaging Initiative

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What Can We Learn From Clinical Trials of Exon Skipping for DMD? door Qi-long Lu, Sebahattin Cirak, Terence Partridge

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Polyquaternium-mediated delivery of morpholino oligonucleotides for exon-skipping in vitro and in mdx mice door Mingxing Wang, Bo Wu, Sapana N Shah, Peijuan Lu, Qilong Lu

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Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy door Joshua J.A. Lee, Rika Maruyama, William Duddy, Hidetoshi Sakurai, Toshifumi Yokota

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Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice door Flavien Bizot, Remko Goossens, Thomas Tensorer, Sergei Dmitriev, Luis Garcia, Annemieke Aartsma-Rus, Pietro Spitali, Aurélie Goyenvalle

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Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1 door Naoko Shiba, Xiao Yang, Mitsuto Sato, Shin Kadota, Yota Suzuki, Masahiro Agata, Kohei Nagamine, Masaki Izumi, Yusuke Honda, Tomoya Koganehira, Hideki Kobayashi, Hajime Ichimura, Shinichiro Chuma, Junichi Nakai, Shugo Tohyama, Keiichi Fukuda, Daigo Miyazaki, Akinori Nakamura, Yuji Shiba

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Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency door HaiFang Yin, Prisca Boisguerin, Hong M. Moulton, Corinne Betts, Yiqi Seow, Jordan Boutilier, Qingsong Wang, Anthony Walsh, Bernard Lebleu, Matthew J.A. Wood

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Context Dependent Effects of Chimeric Peptide Morpholino Conjugates Contribute to Dystrophin Exon-skipping Efficiency door HaiFang Yin, Prisca Boisguerin, Hong M Moulton, Corinne Betts, Yiqi Seow, Jordan Boutilier, Qingsong Wang, Anthony Walsh, Bernard Lebleu, Matthew JA Wood

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

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Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy door Silvana M G Jirka, Christa L Tanganyika-de Winter, Joke W Boertje-van der Meulen, Maaike van Putten, Monika Hiller, Rick Vermue, Peter C de Visser, Annemieke Aartsma-Rus

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Highly efficient CRISPR/Cas9‐mediated exon skipping for recessive dystrophic epidermolysis bullosa door Alex du Rand, John Hunt, Christopher Samson, Evert Loef, Chloe Malhi, Sarah Meidinger, Chun‐Jen Jennifer Chen, Ashley Nutsford, John Taylor, Rod Dunbar, Diana Purvis, Vaughan Feisst, Hilary Sheppard

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Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy For Tauopathies door Reeteka Sud, Evan T Geller, Gerard D Schellenberg

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Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy for Tauopathies

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