Exon skipping to therapeutically restore the frame of an out-of-frame dystrophin mutation has taken center stage in drug development for DMD. The research reviewed here focuses on the …

Antisense oligonucleotide-mediated exon skipping is a promising therapy for DMD. Exon skipping aims to convert out-of-frame mRNA to in-frame mRNA and induce the production of internally …

Antisense oligonucleotide (AON)‐mediated exon skipping is an emerging therapeutic for individuals with Duchenne muscular dystrophy (DMD). Skipping of exons adjacent to common …

1 天前 · Most DMD patients lack full-length dystrophins only because deletions and duplications cluster in hotspot regions encompassing exons 45–55 and 3–9, respectively with ~47% and …

2021年2月18日 · Duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads to difficulties with movement and, eventually, to the need for assisted...

Objective: Exon-skipping therapies aim to convert Duchenne muscular dystrophy (DMD) into less severe Becker muscular dystrophy (BMD) by altering pre-mRNA splicing to restore an open …

2023年6月28日 · Existing exon-skipping therapies work to mask specific exons in the DMD gene sequence using short strands of nucleic acids (which make up DNA and RNA) called …

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 …

2024年10月1日 · Among the various mutations observed along the DMD gene, the deletion of exons 45–55 (del45–55) has been proposed as a promising therapeutic model with the …

2023年9月4日 · Antisense oligonucleotide (AON) mediated exon skipping to restore disrupted dystrophin reading frame is a therapeutic approach that allows production of a shorter but …