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Ryszard Kole

University of North Carolina at Chapel Hill

96

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A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome.

2021 Nature Medicine
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Enhanced delivery of peptide-morpholino oligonucleotides with a small molecule to correct splicing defects in the lung.

2021 Nucleic Acids Research
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Engineered U7 snRNA restores correct β-globin pre-mRNA splicing in mouse βIVS2-654- thalassemic erythroid progenitor cells.

2020 Human Gene Therapy
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Restoration of correct β IVS2-654 -globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells

2019 Scientific Reports
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Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients

2018 Biochemical and Biophysical Research Communications
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Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

2017 Proceedings of the National Academy of Sciences of the United States of America
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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

2017 Journal of Controlled Release
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Exon skipping therapy for Duchenne muscular dystrophy.

2015 Advanced Drug Delivery Reviews
- Ryszard Kole
- Arthur M. Krieg
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Splicing: Hear and Now!

2013 Molecular Therapy
- Ryszard Kole
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snRNA progenitor cells following lentiviral vector delivery of an antisense High-level expression of hemoglobin A in human thalassemic erythroid

2013
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