RNA therapeutics: beyond RNA interference and antisense oligonucleotides
Kole, Ryszard, Krainer, Adrian R., Altman, Sidney
Published in Nature reviews. Drug discovery (01.02.2012)
Published in Nature reviews. Drug discovery (01.02.2012)
Get full text
Journal Article
A targeted antisense therapeutic approach for Hutchinson–Gilford progeria syndrome
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.
Published in Nature medicine (01.03.2021)
Published in Nature medicine (01.03.2021)
Get full text
Journal Article
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
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
Published in The Lancet (British edition) (13.08.2011)
Published in The Lancet (British edition) (13.08.2011)
Get full text
Journal Article
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
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
Published in Lancet neurology (01.10.2009)
Published in Lancet neurology (01.10.2009)
Get full text
Journal Article
Anti-tumor activity of splice-switching oligonucleotides
Bauman, John A, Li, Shyh-Dar, Yang, Angela, Huang, Leaf, Kole, Ryszard
Published in Nucleic acids research (01.12.2010)
Published in Nucleic acids research (01.12.2010)
Get full text
Journal Article
Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice
Jearawiriyapaisarn, Natee, Moulton, Hong M, Buckley, Brian, Roberts, Jennifer, Sazani, Peter, Fucharoen, Suthat, Iversen, Patrick L, Kole, Ryszard
Published in Molecular therapy (01.09.2008)
Published in Molecular therapy (01.09.2008)
Get full text
Journal Article
Restoration of correct βIVS2-654-globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells
Nualkaew, Tiwaporn, Jearawiriyapaisarn, Natee, Hongeng, Suradej, Fucharoen, Suthat, Kole, Ryszard, Svasti, Saovaros
Published in Scientific reports (21.05.2019)
Published in Scientific reports (21.05.2019)
Get full text
Journal Article
Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping
Goyenvalle, Aurélie, Babbs, Arran, Powell, Dave, Kole, Ryszard, Fletcher, Sue, Wilton, Steve D, Davies, Kay E
Published in Molecular therapy (01.01.2010)
Published in Molecular therapy (01.01.2010)
Get full text
Journal Article
Efficient and Persistent Splice Switching by Systemically Delivered LNA Oligonucleotides in Mice
Roberts, Jennifer, Palma, Enzo, Sazani, Peter, Ørum, Henrik, Cho, Moo, Kole, Ryszard
Published in Molecular therapy (01.10.2006)
Published in Molecular therapy (01.10.2006)
Get full text
Journal Article
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
Svasti, Saovaros, Suwanmanee, Thipparat, Fucharoen, Suthat, Moulton, Hong M, Nelson, Michelle H, Maeda, Nobuyo, Smithies, Oliver, Kole, Ryszard
Published in Proceedings of the National Academy of Sciences - PNAS (27.01.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (27.01.2009)
Get full text
Journal Article
Dystromirs as serum biomarkers for monitoring the disease severity in Duchenne muscular Dystrophy
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
Published in PloS one (25.11.2013)
Published in PloS one (25.11.2013)
Get full text
Journal Article
Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids
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
Published in Proceedings of the National Academy of Sciences - PNAS (09.09.2008)
Published in Proceedings of the National Academy of Sciences - PNAS (09.09.2008)
Get full text
Journal Article
Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs
Sazani, P, Kang, S H, Maier, M A, Wei, C, Dillman, J, Summerton, J, Manoharan, M, Kole, R
Published in Nucleic acids research (01.10.2001)
Published in Nucleic acids research (01.10.2001)
Get full text
Journal Article
Restoration of Hemoglobin a Synthesis in Erythroid Cells from Peripheral Blood of Thalassemic Patients
Lacerra, Giuseppina, Sierakowska, Halina, Carestia, Clementina, Fucharoen, Suthat, Summerton, James, Weller, Dwight, Kole, Ryszard
Published in Proceedings of the National Academy of Sciences - PNAS (15.08.2000)
Published in Proceedings of the National Academy of Sciences - PNAS (15.08.2000)
Get full text
Journal Article
Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene
Fletcher, Sue, Adkin, Carl F., Meloni, Penny, Wong, Brenda, Muntoni, Francesco, Kole, Ryszard, Fragall, Clayton, Greer, Kane, Johnsen, Russell, Wilton, Steve D.
Published in Molecular therapy. Nucleic acids (01.10.2012)
Published in Molecular therapy. Nucleic acids (01.10.2012)
Get full text
Journal Article