Double-Target Antisense U7 snRNAs Promote Efficient Skipping of an Aberrant Exon in Three Human β-Thalassemic Mutations

Autor: Linda Gorman, Reto Tomasini, Daniel Suter, Ueli Reber, Daniel Schümperli, Ryszard Kole

Publikováno v: Suter, Daniel; Tomasini, Reto; Reber, Ueli; Gorman, Linda; Kole, Ryszard; Schümperli, Daniel (1999). Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human β-thalassemic mutations. Human molecular genetics, 8(13), pp. 2415-2423. Oxford University Press 10.1093/hmg/8.13.2415

We have used three beta-thalassemic mutations, IVS2-654, -705 and -745, that create aberrant 5' splice sites (5' ss) and activate a common cryptic 3' ss further upstream in intron 2 of the human beta-globin gene to optimize a generally applicable exo

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