Indicated for patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, the drug could be used in around 13% of the disease population. Sarepta’s share price almost ...

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The DMD gene is made up of 79 exons, and mutations in that code can result in a dystrophin deficiency, which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch the ...

Although exon skipping strategies have been applied almost exclusively for dystrophin mutations, they might also be applicable for other genetic disorders where protein truncation is compatible ...

Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...

CONNECT1 has enrolled two cohorts of boys and young men living with DMD amenable to exon 51 skipping and its endpoints include safety and tolerability, dystrophin production, exon skipping ...

Gene therapies, such as CRISPR/Cas9-mediated gene editing, exon skipping, and antisense oligonucleotides, aim to restore dystrophin expression but face challenges in effectively targeting MuSCs.

Intronic primers flanking exon 9 were designed using the PAC sequence available in the Genbank sequence database (clone DJ0054D12; accession number AC006342). PCR amplification of an approximate 6 ...