Alternative splicing (AS) of pre-mRNA plays a crucial role in tissue-specific gene regulation, with disease implications due to splicing defects. Predicting and manipulating AS can therefore uncover ...

Precision BioSciences (DTIL) announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of ...

The company plans to submit a Biologics Licence Application to the US Food and Drug Administration later in 2025 for its DMD ...

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...

The diagnosis of Duchenne muscular dystrophy was made in all patients on the basis of the clinical history and the absence or near-absence of the dystrophin protein on muscle biopsy. In addition ...

63% of mice treated with two rAAV9-microdystrophin doses produced antibodies to dystrophin, which was less in mice treated with two rAAV9-microdystrophin doses and combination therapy (25%). Likewise, ...

Patients treated in Part 1 of the study had biopsies taken at 64 weeks after dosing and showed consistent and sustained expression of ELEVIDYS micro-dystrophin compared to week 12 biopsies ...

About EXONDYS 51 EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in ...

The goal of exon-skipping therapy is to get cells to skip over one or more specific exons when the gene is used to make dystrophin, thus letting cells make a shorter but functional version of the ...

Large Introns suggest the presence of transcriptional elements and Exons that exhibit interspecies variations, similar to the Dystrophin gene. In silico analysis of the Introns within the PRKN gene ...

Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMD Date and Time ...