RNA therapeutics: beyond RNA interference and antisense oligonucleotides von Kole, Ryszard, Krainer, Adrian R., Altman, Sidney

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A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome von Erdos, Michael R., Cabral, Wayne A., Tavarez, Urraca L., Cao, Kan, Gvozdenovic-Jeremic, Jelena, Narisu, Narisu, Zerfas, Patricia M., Crumley, Stacy, Boku, Yoseph, Hanson, Gunnar, Mourich, Dan V., Kole, Ryszard, Eckhaus, Michael A., Gordon, Leslie B., Collins, Francis S.

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Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-... von Cirak, Sebahattin, MD, Arechavala-Gomeza, Virginia, PhD, Guglieri, Michela, MD, Feng, Lucy, PhD, Torelli, Silvia, PhD, Anthony, Karen, PhD, Abbs, Stephen, PhD, Garralda, Maria Elena, Prof, Bourke, John, MD, Wells, Dominic J, Prof, Dickson, George, Prof, Wood, Matthew JA, MD PhD, Wilton, Steve D, Prof, Straub, Volker, Prof, Kole, Ryszard, Prof, Shrewsbury, Stephen B, MD, Sewry, Caroline, Prof, Morgan, Jennifer E, PhD, Bushby, Kate, Prof, Muntoni, Francesco, Prof

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Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing von Sazani, Peter, Kole, Ryszard

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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-conce... von Kinali, Maria, MD, Arechavala-Gomeza, Virginia, PhD, Feng, Lucy, PhD, Cirak, Sebahattin, MD, Hunt, David, FRCS, Adkin, Carl, PhD, Guglieri, Michela, MD, Ashton, Emma, PhD, Abbs, Stephen, PhD, Nihoyannopoulos, Petros, MD, Garralda, Maria Elena, MD, Rutherford, Mary, MD, Mcculley, Caroline, PhD, Popplewell, Linda, PhD, Graham, Ian R, PhD, Dickson, George, PhD, Wood, Matthew JA, MD, Wells, Dominic J, PhD, Wilton, Steve D, PhD, Kole, Ryszard, PhD, Straub, Volker, MD, Bushby, Kate, MD, Sewry, Caroline, PhD, Morgan, Jennifer E, PhD, Muntoni, Francesco, MD

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Anti-tumor activity of splice-switching oligonucleotides von Bauman, John A, Li, Shyh-Dar, Yang, Angela, Huang, Leaf, Kole, Ryszard

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Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice von Jearawiriyapaisarn, Natee, Moulton, Hong M, Buckley, Brian, Roberts, Jennifer, Sazani, Peter, Fucharoen, Suthat, Iversen, Patrick L, Kole, Ryszard

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Restoration of correct βIVS2-654-globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells von Nualkaew, Tiwaporn, Jearawiriyapaisarn, Natee, Hongeng, Suradej, Fucharoen, Suthat, Kole, Ryszard, Svasti, Saovaros

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Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping von Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E

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Efficient and Persistent Splice Switching by Systemically Delivered LNA Oligonucleotides in Mice von Roberts, Jennifer, Palma, Enzo, Sazani, Peter, Ørum, Henrik, Cho, Moo, Kole, Ryszard

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Cellular Response to an Antisense-mediated Shift of Bcl-x Pre-mRNA Splicing and Antineoplastic Agents von Mercatante, Danielle R., Mohler, James L., Kole, Ryszard

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RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice von Svasti, Saovaros, Suwanmanee, Thipparat, Fucharoen, Suthat, Moulton, Hong M, Nelson, Michelle H, Maeda, Nobuyo, Smithies, Oliver, Kole, Ryszard

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Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy von Zaharieva, Irina T, Calissano, Mattia, Scoto, Mariacristina, Preston, Mark, Cirak, Sebahattin, Feng, Lucy, Collins, James, Kole, Ryszard, Guglieri, Michela, Straub, Volker, Bushby, Kate, Ferlini, Alessandra, Morgan, Jennifer E, Muntoni, Francesco

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Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids von Chin, Joanna Y, Kuan, Jean Y, Lonkar, Pallavi S, Krause, Diane S, Seidman, Michael M, Peterson, Kenneth R, Nielsen, Peter E, Kole, Ryszard, Glazer, Peter M

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Exon skipping therapy for Duchenne muscular dystrophy von Kole, Ryszard, Krieg, Arthur M.

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Peptide-Morpholino Conjugate: A Promising Therapeutic for Duchenne Muscular Dystrophy von Moulton, Hong M., Wu, Bo, Jearawiriyapaisarn, Natee, Sazani, Peter, Lu, Qi Long, Kole, Ryszard

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Applications of the PMO platform to genetic diseases von Kole, Ryszard

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Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs von Sazani, P, Kang, S H, Maier, M A, Wei, C, Dillman, J, Summerton, J, Manoharan, M, Kole, R

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Self-aggregating 1.8 kDa polyethylenimines with dissolution switch at endosomal acidic pH are delivery carriers for plasmid DNA, mRNA, siRNA and exon-skipping oligonucleotides von Chiper, Manuela, Tounsi, Nassera, Kole, Ryszard, Kichler, Antoine, Zuber, Guy

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Modulation of RNA splicing as a potential treatment for cancer von Bauman, John A., Kole, Ryszard

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