Year |
Citation |
Score |
2020 |
d'Arqom A, Nualkaew T, Jearawiriyapaisarn N, Kole R, Svasti S. Engineered U7 snRNA restores correct β-globin pre-mRNA splicing in mouse βIVS2-654- thalassemic erythroid progenitor cells. Human Gene Therapy. PMID 32977730 DOI: 10.1089/hum.2020.145 |
0.341 |
|
2013 |
Kole R. Splicing: hear and now! Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 920-1. PMID 23636290 DOI: 10.1038/Mt.2013.75 |
0.4 |
|
2012 |
Fletcher S, Adkin CF, Meloni P, Wong B, Muntoni F, Kole R, Fragall C, Greer K, Johnsen R, Wilton SD. Targeted exon skipping to address "leaky" mutations in the dystrophin gene. Molecular Therapy. Nucleic Acids. 1: e48. PMID 23344648 DOI: 10.1038/mtna.2012.40 |
0.359 |
|
2012 |
Kole R, Krainer AR, Altman S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nature Reviews. Drug Discovery. 11: 125-40. PMID 22262036 DOI: 10.1038/Nrd3625 |
0.306 |
|
2011 |
Fragall CT, Adams AM, Johnsen RD, Kole R, Fletcher S, Wilton SD. Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching. Bmc Medical Genetics. 12: 141. PMID 22013876 DOI: 10.1186/1471-2350-12-141 |
0.328 |
|
2011 |
Bauman JA, Kole R. Modulation of RNA splicing as a potential treatment for cancer. Bioengineered Bugs. 2: 125-8. PMID 21637003 DOI: 10.4161/Bbug.2.3.15165 |
0.317 |
|
2010 |
Goyenvalle A, Babbs A, Powell D, Kole R, Fletcher S, Wilton SD, Davies KE. Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 198-205. PMID 19844193 DOI: 10.1038/mt.2009.248 |
0.302 |
|
2009 |
Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, ... ... Kole R, et al. 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. The Lancet. Neurology. 8: 918-28. PMID 19713152 DOI: 10.1016/S1474-4422(09)70211-X |
0.316 |
|
2009 |
Lonkar P, Kim KH, Kuan JY, Chin JY, Rogers FA, Knauert MP, Kole R, Nielsen PE, Glazer PM. Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids. Nucleic Acids Research. 37: 3635-44. PMID 19364810 DOI: 10.1093/Nar/Gkp217 |
0.48 |
|
2009 |
Svasti S, Suwanmanee T, Fucharoen S, Moulton HM, Nelson MH, Maeda N, Smithies O, Kole R. RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice. Proceedings of the National Academy of Sciences of the United States of America. 106: 1205-10. PMID 19164558 DOI: 10.1073/Pnas.0812436106 |
0.583 |
|
2009 |
Bauman J, Jearawiriyapaisarn N, Kole R. Therapeutic potential of splice-switching oligonucleotides. Oligonucleotides. 19: 1-13. PMID 19125639 DOI: 10.1089/Oli.2008.0161 |
0.332 |
|
2008 |
Chin JY, Kuan JY, Lonkar PS, Krause DS, Seidman MM, Peterson KR, Nielsen PE, Kole R, Glazer PM. Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids. Proceedings of the National Academy of Sciences of the United States of America. 105: 13514-9. PMID 18757759 DOI: 10.1073/Pnas.0711793105 |
0.518 |
|
2007 |
Resina S, Kole R, Travo A, Lebleu B, Thierry AR. Switching on transgene expression by correcting aberrant splicing using multi-targeting steric-blocking oligonucleotides. The Journal of Gene Medicine. 9: 498-510. PMID 17471591 DOI: 10.1002/Jgm.1044 |
0.313 |
|
2006 |
Roberts J, Palma E, Sazani P, Ørum H, Cho M, Kole R. Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 14: 471-5. PMID 16854630 DOI: 10.1016/J.Ymthe.2006.05.017 |
0.306 |
|
2004 |
Kole R, Williams T, Cohen L. RNA modulation, repair and remodeling by splice switching oligonucleotides. Acta Biochimica Polonica. 51: 373-8. PMID 15218534 DOI: 10.18388/Abp.2004_3576 |
0.379 |
|
2004 |
Asparuhova M, Kole R, Schumperli D. Antisense derivatives of U7 and other small nuclear RNAs as tools to modify pre-mRNA splicing patterns Gene Therapy and Regulation. 2: 321-349. DOI: 10.1163/1568558043967472 |
0.301 |
|
2003 |
Vacek MM, Ma H, Gemignani F, Lacerra G, Kafri T, Kole R. High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA. Blood. 101: 104-11. PMID 12393543 DOI: 10.1182/Blood-2002-06-1869 |
0.564 |
|
2002 |
Suwanmanee T, Sierakowska H, Fucharoen S, Kole R. Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 718-26. PMID 12498768 DOI: 10.1006/Mthe.2002.0805 |
0.517 |
|
2002 |
Sazani P, Gemignani F, Kang SH, Maier MA, Manoharan M, Persmark M, Bortner D, Kole R. Systemically delivered antisense oligomers upregulate gene expression in mouse tissues. Nature Biotechnology. 20: 1228-33. PMID 12426578 DOI: 10.1038/Nbt759 |
0.334 |
|
2002 |
Gemignani F, Sazani P, Morcos P, Kole R. Temperature-dependent splicing of beta-globin pre-mRNA. Nucleic Acids Research. 30: 4592-8. PMID 12409448 DOI: 10.1093/Nar/Gkf607 |
0.458 |
|
2002 |
Suwanmanee T, Sierakowska H, Lacerra G, Svasti S, Kirby S, Walsh CE, Fucharoen S, Kole R. Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides. Molecular Pharmacology. 62: 545-53. PMID 12181431 DOI: 10.1124/Mol.62.3.545 |
0.542 |
|
2001 |
Mercatante DR, Sazani P, Kole R. Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases. Current Cancer Drug Targets. 1: 211-30. PMID 12188880 |
0.342 |
|
2001 |
Kole R, Sazani P. Antisense effects in the cell nucleus: Modification of splicing Current Opinion in Molecular Therapeutics. 3: 229-234. PMID 11497345 |
0.357 |
|
2000 |
Gorman L, Mercatante DR, Kole R. Restoration of correct splicing of thalassemic beta-globin pre-mRNA by modified U1 snRNAs. The Journal of Biological Chemistry. 275: 35914-9. PMID 10969081 DOI: 10.1074/Jbc.M006259200 |
0.489 |
|
2000 |
Sierakowska H, Gorman L, Kang SH, Kole R. Antisense oligonucleotides and RNAs as modulators of pre-mRNA splicing Methods in Enzymology. 313: 506-521. PMID 10595376 DOI: 10.1016/S0076-6879(00)13032-0 |
0.418 |
|
1999 |
Friedman KJ, Kole J, Cohn JA, Knowles MR, Silverman LM, Kole R. Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides Journal of Biological Chemistry. 274: 36193-36199. PMID 10593905 DOI: 10.1074/Jbc.274.51.36193 |
0.389 |
|
1999 |
Suter D, Tomasini R, Reber U, Gorman L, Kole R, Schümperli D. Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human β-thalassemic mutations Human Molecular Genetics. 8: 2415-2423. PMID 10556289 DOI: 10.1093/Hmg/8.13.2415 |
0.367 |
|
1999 |
Wilton SD, Lloyd F, Carville K, Fletcher S, Honeyman K, Agrawal S, Kole R. Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides Neuromuscular Disorders. 9: 330-338. PMID 10407856 DOI: 10.1016/S0960-8966(99)00010-3 |
0.405 |
|
1999 |
Sierakowska H, Sambade MJ, Schümperli D, Kole R. Sensitivity of splice sites to antisense oligonucleotides in vivo Rna. 5: 369-377. PMID 10094306 DOI: 10.1017/S135583829998130X |
0.341 |
|
1998 |
Kang SH, Cho MJ, Kole R. Up-regulation of luciferase gene expression with antisense oligonucleotides: Implications and applications in functional assay development Biochemistry. 37: 6235-6239. PMID 9572837 DOI: 10.1021/Bi980300H |
0.348 |
|
1998 |
Gorman L, Suter D, Emerick V, Schümperli D, Kole R. Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs Proceedings of the National Academy of Sciences of the United States of America. 95: 4929-4934. PMID 9560205 DOI: 10.1073/Pnas.95.9.4929 |
0.316 |
|
1998 |
Lewis J, Yang B, Kim R, Sierakowska H, Kole R, Smithies O, Maeda N. A common human beta globin splicing mutation modeled in mice. Blood. 91: 2152-6. PMID 9490703 DOI: 10.1182/Blood.V91.6.2152.2152_2152_2156 |
0.46 |
|
1997 |
Kole R. Modification of pre-mRNA splicing by antisense oligonucleotides Acta Biochimica Polonica. 44: 231-238. PMID 9360712 |
0.363 |
|
1996 |
Sierakowska H, Sambade MJ, Agrawal S, Kole R. Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides Proceedings of the National Academy of Sciences of the United States of America. 93: 12840-12844. PMID 8917506 DOI: 10.1073/Pnas.93.23.12840 |
0.309 |
|
1993 |
Dominski Z, Kole R. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides Proceedings of the National Academy of Sciences of the United States of America. 90: 8673-8677. PMID 8378346 DOI: 10.1073/Pnas.90.18.8673 |
0.379 |
|
1992 |
Dominski Z, Kole R. Cooperation of pre-mRNA sequence elements in splice site selection Molecular and Cellular Biology. 12: 2108-2114. PMID 1569943 DOI: 10.1128/Mcb.12.5.2108 |
0.309 |
|
1991 |
Dominski Z, Kole R. Selection of Splice Sites in Pre-mRNAs with Short Internal Exons Molecular and Cellular Biology. 11: 6075-6083. PMID 1944277 DOI: 10.1128/Mcb.11.12.6075 |
0.345 |
|
1989 |
Sierakowska H, Shukla RR, Dominski Z, Kole R. Inhibition of pre-mRNA splicing by 5-fluoro-, 5-chloro-, and 5-bromouridine Journal of Biological Chemistry. 264: 19185-19191. PMID 2530228 |
0.303 |
|
1988 |
Furdon PJ, Kole R. The length of the downstream exon and the substitution of specific sequences affect pre-mRNA splicing in vitro Molecular and Cellular Biology. 8: 860-866. PMID 3352607 DOI: 10.1128/Mcb.8.2.860 |
0.398 |
|
1982 |
Kole R, Weissman SM. Accurate in vitro splicing of human β-globin RNA Nucleic Acids Research. 10: 5429-5445. PMID 6292841 DOI: 10.1093/nar/10.18.5429 |
0.473 |
|
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