2023年9月12日 · We now demonstrate that CRISPR-SKIP can be adapted to correct some forms of Duchenne muscular dystrophy by disrupting the splice acceptor in human DMD exon 45 …

2021年2月25日 · Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients …

2024年10月1日 · Deletion of 45 – 55 DMD exons (del45 – 55) was described in asymptomatic subjects, but recently serious skeletal and cardiac complications have been reported. …

Here, we show the selection of U1 snRNA-antisense constructs able to confer effective rescue of dystrophin synthesis in a Δ44 Duchenne genetic background, through skipping of exon 45; …

AMONDYS 45 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation of the dystrophin gene that can be treated by skipping exon 45. This …

Exon skipping is a mutation-specific approach. Which exon is skipped depends on the size and location of the mutation. For example, Casimersen, an exon 45 skipping molecule, is an …

2023年7月27日 · We now demonstrate that CRISPR-SKIP can be adapted to correct some forms of Duchenne muscular dystrophy by disrupting the splice acceptor in human DMD exon 45 …

The most common single exon deletion-exon 45 (Δ45)-may theoretically be treated by skipping of either flanking exon (44 or 46). We sought to predict the impact of these by assessing the …

We now DMD demonstrate that CRISPR-SKIP can be adapted to correct some forms of Duchenne muscular dystrophy by disrupting the splice acceptor in human exon 45 with high …

2024年10月1日 · Treatment of DMD patient-derived skeletal and cardiac muscle cells harboring an exon 45 amenable mutation deletion demonstrated robust, dose-dependent exon 45 …