<i>DMD</i> antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon.

Goossens, Remko; Verwey, Nisha; Ariyurek, Yavuz; Schnell, Fred; Aartsma-Rus, Annemieke

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon.

Goossens, Remko; Verwey, Nisha; Ariyurek, Yavuz; Schnell, Fred; Aartsma-Rus, Annemieke.

Afiliação

Goossens R; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Verwey N; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Ariyurek Y; Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
Schnell F; Leiden Genome Technology Center, Leiden University Medical Center, Leiden, The Netherlands.
Aartsma-Rus A; Sarepta Therapeutics, Inc, Cambridge, MA, USA.

RNA Biol ; 20(1): 693-702, 2023 01.

Article em En | MEDLINE | ID: mdl-37667454

Assuntos

Distrofia Muscular de Duchenne; Oligonucleotídeos Antissenso; Humanos; Oligonucleotídeos Antissenso/genética; Íntrons; Distrofina/genética; Éxons; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/terapia

Palavras-chave

AON; ASO; Antisense oligonucleotides; DMD; Duchenne Muscular Dystrophy; Dystrophin; Exon skipping

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Oligonucleotídeos Antissenso / Distrofia Muscular de Duchenne Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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