Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping.

McCormack, Nikki M; Calabrese, Kelsey A; Sun, Christina M; Tully, Christopher B; Heier, Christopher R; Fiorillo, Alyson A

McCormack, Nikki M; Calabrese, Kelsey A; Sun, Christina M; Tully, Christopher B; Heier, Christopher R; Fiorillo, Alyson A.

Afiliación

McCormack NM; Center for Genetic Medicine Research, Children's National Hospital, Washington, District of Columbia, USA.
Calabrese KA; Center for Genetic Medicine Research, Children's National Hospital, Washington, District of Columbia, USA.
Sun CM; Center for Genetic Medicine Research, Children's National Hospital, Washington, District of Columbia, USA.
Tully CB; Center for Genetic Medicine Research, Children's National Hospital, Washington, District of Columbia, USA.
Heier CR; Center for Genetic Medicine Research, Children's National Hospital, Washington, District of Columbia, USA.
Fiorillo AA; Department of Genomics and Precision Medicine, George Washington University School of Medicine and Health Sciences, Washington, District of Columbia, USA.

bioRxiv ; 2023 Aug 08.

Article en En | MEDLINE | ID: mdl-37214870

Palabras clave

Becker Muscular Dystrophy (BMD); Duchenne Muscular Dystrophy (DMD); RNA therapeutics; antisense oligonucleotides; dystrophin; exon skipping; miR-146a-5p; microRNA

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: BioRxiv Año: 2023 Tipo del documento: Article País de afiliación: Estados Unidos

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