Beta-thalassemia treated in mice with splice-modifying Morpholino
Jon Moulton
Wednesday, 04 February 2009 16:24 UTC
Morpholinos conjugated with cell-penetrating peptides were injected into mice carrying a splice-site mutation in beta-globin. The Morpholinos blocked the mutant splice sites and restored normal splicing so that the mice produced fully-functional hemoglobin.
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. Proc Natl Acad Sci U S A. 2009 Jan 27;106(4):1205-10. Epub 2009 Jan 21.
http://www.ncbi.nlm.nih.gov/pubmed/19164558
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