The invention relates to antisense oligonucleotides capable of inducing exon skipping in dystrophin gene and their use in treatment of Duchenne muscular dystrophy (DMD). Skipping of said exons from the mRNA dystrophin transcript promotes prodn. of a functional or partially functional dystrophin protein in a patient carrying a small mutation in said exon. Such a use is characterized in that the exon is selected from the group consisting of exon 10, exon 16, exon 26, exon 33 and exon 34 of the dystrophin gene.
Antisense oligonucleotides capable of inducing exon skipping in dystrophin gene and their use in treatment of duchenne muscular dystrophy
FERLINI, Alessandra;MEDICI, Alessandro;PERRONE, Daniela;RIMESSI, Paola;SPITALI, Pietro
2010
Abstract
The invention relates to antisense oligonucleotides capable of inducing exon skipping in dystrophin gene and their use in treatment of Duchenne muscular dystrophy (DMD). Skipping of said exons from the mRNA dystrophin transcript promotes prodn. of a functional or partially functional dystrophin protein in a patient carrying a small mutation in said exon. Such a use is characterized in that the exon is selected from the group consisting of exon 10, exon 16, exon 26, exon 33 and exon 34 of the dystrophin gene.File in questo prodotto:
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