Therapeutic potential of splice-switching oligonucleotides

بواسطة John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole
منشور في 2018

Repair of a splicing defect in erythroid cells from patients with β-thalassemia/HbE disorder

بواسطة Thipparat Suwanmanee, Halina Sierakowska, Suthat Fucharoen, Ryszard Kole
منشور في 2018

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

بواسطة Natee Jearawiriyapaisarn, Hong M. Moulton, Peter Sazani, Ryszard Kole, Monte S. Willis
منشور في 2018

Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β^iVS2-654-Thalassemic Erythroid Progenitor Cells

بواسطة Annette D'Arqom, Tiwaporn Nualkaew, Natee Jearawiriyapaisarn, Ryszard Kole, Saovaros Svasti
منشور في 2022

Peptide-morpholino conjugate: A promising therapeutic for duchenne muscular dystrophy

بواسطة Hong M. Moulton, Bo Wu, Natee Jearawiriyapaisarn, Peter Sazani, Qi Long Lu, Ryszard Kole
منشور في 2018

Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients

بواسطة Giuseppina Lacerra, Halina Sierakowska, Clementina Carestia, Suthat Fucharoen, James Summerton, Dwight Weller, Ryszard Kole
منشور في 2018

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

بواسطة Sarinthip Preedagasamzin, Tiwaporn Nualkaew, Tanjitti Pongrujikorn, Natini Jinawath, Ryszard Kole, Suthat Fucharoen, Natee Jearawiriyapaisarn, Saovaros Svasti
منشور في 2019

Restoration of human β-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides

بواسطة Thipparat Suwanmanee, Halin Sierakowska, Giuseppina Lacerra, Saovaros Svasti, Suzanne Kirby, Christopher E. Walsh, Suthat Fucharoen, Ryszard Kole
منشور في 2018

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice

بواسطة Natee Jearawiriyapaisarn, Hong M. Moulton, Brian Buckley, Jennifer Roberts, Peter Sazani, Suthat Fucharoen, Patrick L. Iversen, Ryszard Kole
منشور في 2018

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

بواسطة Saovaros Svasti, Thipparat Suwanmanee, Suthat Fucharoen, Hong M. Moulton, Michelle H. Nelson, Nobuyo Maeda, Oliver Smithies, Ryszard Kole
منشور في 2018