Molecular Genetic Analysis of a DMD Frameshift Mutation in a Boy with Duchenne Muscular Dystrophy by MLPA and Sanger Sequencing by Chen Q, Zhang W, Zha L

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Understanding Duchenne muscular dystrophy-associated brain pathology by Minou A. T. Verhaeg, Rosanne Govaarts, Maaike van Putten

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Intrinsic Alu affects for RNA splicing in a minigene model by Mina Nakama, Bunta Imanaka, Yuma Kimoto

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MSI expresso: a software for determining MSI status and detecting MSI-related transcription events from RNA sequencing data by Emmanuel Tubacher, Alexandre How-Kit, Mourad Sahbatou, Alex Duval, Victor Renault, Jean-François Deleuze, Jean-François Deleuze

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

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Antisense‐mediated exon skipping: a therapeutic strategy for titin‐based dilated cardiomyopathy by Michael Gramlich, Luna Simona Pane, Qifeng Zhou, Zhifen Chen, Marta Murgia, Sonja Schötterl, Alexander Goedel, Katja Metzger, Thomas Brade, Elvira Parrotta, Martin Schaller, Brenda Gerull, Ludwig Thierfelder, Annemieke Aartsma‐Rus, Siegfried Labeit, John J Atherton, Julie McGaughran, Richard P Harvey, Daniel Sinnecker, Matthias Mann, Karl‐Ludwig Laugwitz, Meinrad Paul Gawaz, Alessandra Moretti

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Genetic technology in the targeted therapy of Alport Syndrome by Magdalena Kłusek, Bartosz Pawłowski, Michał Sienkiewicz, Anna Mandziuk

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Possibility of exon skipping therapy for Duchenne muscular dystrophy in Russian patients: present and future by E. V. Zinina, M. V. Bulakh, O. P. Ryzhkova, O. A. Shchagina, A. V. Polyakov

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Modern methods of therapy of Duchenne muscular dystrophy: literature review with a clinical case by S. B. Artemyeva, О. А. Shidlovskaya, Yu. О. Papina, А. V. Monakhova, I. V. Shulyakov, D. V. Vlodavets

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Identified five variants in CFTR gene that alter RNA splicing by minigene assay by Bingying Zhang, Bingying Zhang, Yiyin Zhang, Yan Zhang, Xuyan Liu, Ran Zhang, Zhi Wang, Fengjiao Pan, Ning Xu, Leping Shao

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Unlocking the therapeutic potential of RNA splicing in lung fibrosis: Insights from the SRSF7–PKM axis by Bi-Sen Ding

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Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice by Christina Gedicke‐Hornung, Verena Behrens‐Gawlik, Silke Reischmann, Birgit Geertz, Doreen Stimpel, Florian Weinberger, Saskia Schlossarek, Guillaume Précigout, Ingke Braren, Thomas Eschenhagen, Giulia Mearini, Stéphanie Lorain, Thomas Voit, Patrick A. Dreyfus, Luis Garcia, Lucie Carrier

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Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis by Shannon Grabich, Brian Ung, Aalok Nadkar, Kathryn DeYoung, James Signorovitch, Aravindhan Veerapandiyan

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Advances in Nucleic Acid Drugs and Gene Therapies based on Animal Models of Duchenne Muscular Dystrophy by LIU Siyu, LAI Yuezhao, GUO Wenting, CHEN Xuejin

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Novel MSH6 exon 5–6 skipping variant in a Taiwanese family with Lynch syndrome: implications for genetic testing and cancer management by Ting-Yao Wang, Chao-Yu Chen, Huei-Chieh Chuang, Yuan-Yuan Jiang, Jrhau Lung

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Rescue of a panel of Hemophilia A-causing 5’ss splicing mutations by unique Exon-specific U1snRNA variants by Laura Peretto, Claudia D’angiolillo, Paolo Ferraresi, Dario Balestra, Mirko Pinotti

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Splice-site mutation enhances the efficiency of CRISPR-mediated gene knockout and new germplasm creation in fish by Zhen Xu, Ma Zhuo, Leihui Wang, Shuo Yan, Shiyi Zhang, Ying Chen, Guang Xu, Chenxu Wang, Wenjing Tao, Deshou Wang

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When HERG-caused LQT2 encounters antisense oligonucleotide: is exon 6 skipping therapy plausible? by Zequn Zheng, Zequn Zheng, Yongfei Song

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