Ryzard Kole
Affiliations: | Pharmacology | University of North Carolina, Chapel Hill, Chapel Hill, NC |
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| d'Arqom A, Nualkaew T, Jearawiriyapaisarn N, et al. (2020) Engineered U7 snRNA restores correct β-globin pre-mRNA splicing in mouse βIVS2-654- thalassemic erythroid progenitor cells. Human Gene Therapy |
| Kole R. (2013) Splicing: hear and now! Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 920-1 |
| Fletcher S, Adkin CF, Meloni P, et al. (2012) Targeted exon skipping to address "leaky" mutations in the dystrophin gene. Molecular Therapy. Nucleic Acids. 1: e48 |
| Kole R, Krainer AR, Altman S. (2012) RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nature Reviews. Drug Discovery. 11: 125-40 |
| Fragall CT, Adams AM, Johnsen RD, et al. (2011) Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching. Bmc Medical Genetics. 12: 141 |
| Bauman JA, Kole R. (2011) Modulation of RNA splicing as a potential treatment for cancer. Bioengineered Bugs. 2: 125-8 |
| Goyenvalle A, Babbs A, Powell D, et al. (2010) 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 |
| Kinali M, Arechavala-Gomeza V, Feng L, et al. (2009) 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 |
| Lonkar P, Kim KH, Kuan JY, et al. (2009) Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids. Nucleic Acids Research. 37: 3635-44 |
| Svasti S, Suwanmanee T, Fucharoen S, et al. (2009) 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 |