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.
2010
File in questo prodotto:
Non ci sono file associati a questo prodotto.

I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11392/1823703
 Attenzione

Attenzione! I dati visualizzati non sono stati sottoposti a validazione da parte dell'ateneo

Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus ND
  • ???jsp.display-item.citation.isi??? ND
social impact