Upper Limb Changes in DMD Patients Amenable to Skipping Exons 44, 45, 51 and 53: A 24-Month Study by Claudia Brogna, Marika Pane, Giorgia Coratti, Adele D’Amico, Elena Pegoraro, Luca Bello, Valeria Ada Maria Sansone, Emilio Albamonte, Sonia Messina, Antonella Pini, Maria Grazia D’Angelo, Claudio Bruno, Tiziana Mongini, Federica Silvia Ricci, Angela Berardinelli, Roberta Battini, Riccardo Masson, Enrico Silvio Bertini, Luisa Politano, Eugenio Mercuri, Italian DMD Group

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Novel splice-affecting variants in CYP27A1 gene in two Chilean patients with Cerebrotendinous Xanthomatosis by Susan V. Smalley, Yudith Preiss, José Suazo, Javier Andrés Vega, Isidora Angellotti, Carlos F. Lagos, Enzo Rivera, Karin Kleinsteuber, Javier Campion, J. Alfredo Martínez, Alberto Maiz, José Luis Santos

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Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides by Sofia Karkampouna, Boudewijn PT Kruithof, Peter Kloen, Miryam C Obdeijn, Annelies MA van der Laan, Hans J Tanke, Dwi U Kemaladewi, Willem MH Hoogaars, Peter AC 't Hoen, Annemieke Aartsma-Rus, Ian M Clark, Peter ten Dijke, Marie-José Goumans, Marianna Kruithof-de Julio

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A survey of computational methods in transcriptome-wide alternative splicing analysis by Wang Jianbo, Ye Zhenqing, Huang Tim H.-M., Shi Huidong, Jin Victor

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CRISPR-induced exon skipping is dependent on premature termination codon mutations by Tingting Sui, Yuning Song, Zhiquan Liu, Mao Chen, Jichao Deng, Yuanyuan Xu, Liangxue Lai, Zhanjun Li

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Analysis of Exon Skipping Applicability for Dysferlinopathies by Jamie Leckie, Sebastian Hernandez Rodriguez, Martin Krahn, Toshifumi Yokota

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Human CD72 splicing isoform responsible for resistance to systemic lupus erythematosus regulates serum immunoglobulin level and is localized in endoplasmic reticulum by Hitomi Yuki, Adachi Takahiro, Tsuchiya Naoyuki, Honda Zen-Ichiro, Tokunaga Katsushi, Tsubata Takeshi

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Synthesis of a Morpholino Nucleic Acid (MNA)-Uridine Phosphoramidite, and Exon Skipping Using MNA/2′-O-Methyl Mixmer Antisense Oligonucleotide by Suxiang Chen, Bao T. Le, Kamal Rahimizadeh, Khalil Shaikh, Narinder Mohal, Rakesh N. Veedu

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CRISPR/Cas9 disruption of EpCAM Exon 2 results in cell-surface expression of a truncated protein targeted by an EpCAM specific T cell engager by Andisheh Bagheri, Patricia A. Culp, Robert B. DuBridge, Tseng-hui Timothy Chen

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Splicing mutations in AMELX and ENAM cause amelogenesis imperfecta by Zhenwei Zhang, Xiaoying Zou, Lin Feng, Yu Huang, Feng Chen, Kai Sun, Yilin Song, Ping Lv, Xuejun Gao, Yanmei Dong, Hua Tian

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The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery by Agathe Eckenfelder, Julie Tordo, Arran Babbs, Kay E Davies, Aurélie Goyenvalle, Olivier Danos

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Modeling Patient-Specific Muscular Dystrophy Phenotypes and Therapeutic Responses in Reprogrammed Myotubes Engineered on Micromolded Gelatin Hydrogels by Florian Barthélémy, Florian Barthélémy, Jeffrey W. Santoso, Laura Rabichow, Laura Rabichow, Rongcheng Jin, Isaiah Little, Isaiah Little, Stanley F. Nelson, Stanley F. Nelson, Stanley F. Nelson, Megan L. McCain, Megan L. McCain, M. Carrie Miceli, M. Carrie Miceli

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Fructose Promotes Uptake and Activity of Oligonucleotides With Different Chemistries in a Context-dependent Manner in mdx Mice by Limin Cao, Gang Han, Caorui Lin, Ben Gu, Xianjun Gao, Hong M Moulton, Yiqi Seow, HaiFang Yin

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Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle by Camilla Brolin, Takehiko Shiraishi, Pernille Hojman, Thomas O Krag, Peter E Nielsen, Julie Gehl

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Ninein, a candidate gene for ethanol anxiolysis, shows complex exon-specific expression and alternative splicing differences between C57BL/6J and DBA/2J mice by E. R. Gnatowski, E. R. Gnatowski, J. L. Jurmain, M. G. Dozmorov, M. G. Dozmorov, J. T. Wolstenholme, J. T. Wolstenholme, M. F. Miles, M. F. Miles

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Poly(ester amine) Composed of Polyethylenimine and Pluronic Enhance Delivery of Antisense Oligonucleotides In Vitro and in Dystrophic mdx Mice by Mingxing Wang, Bo Wu, Jason D Tucker, Lauren E Bollinger, Peijuan Lu, Qilong Lu

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Efficacy and safety of Viltepso® in Duchenne muscular dystrophy: review of clinical studies by V. M. Suslov, D. I. Rudenko

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MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice by Ning Ran, Caorui Lin, Ling Leng, Gang Han, Mengyuan Geng, Yingjie Wu, Scott Bittner, Hong M Moulton, HaiFang Yin

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Endometrial gland-specific progestagenassociated endometrial protein and cilia gene splicing changes in recurrent pregnancy loss by Jennifer E Pearson-Farr, Gabrielle Wheway, Maaike S A Jongen, Patricia Goggin, Rohan M Lewis, Ying Cheong, Jane K Cleal

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Experience of observing patients with Duchenne myopathy by L. I. Minaycheva, E. Yu. Petlina, E. G. Ravzhaeva, G. N. Seitova

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