Gene Therapy for Bardet-Biedl Syndrome

Gene Therapy for Duchenne Muscular DystrophyExon skipping approach based on the employment of Antisense oligonucleotide (PMOs and PPMOs) combined with different approved drugs modulating the UPR (unfolded protein response), UPS (ubiquitin proteasome system) and chaperone system.In this in vitro study, DMD patient derived cells are treated with specific PMOs and PPMOs to induce skipping of certain exons in the DMD pre-mRNA and produce dystrophin which levels could be increased by simultaneous treatment with approved modulating drugs.Lab techniques: cell culture, toxicological assays (MTT assay), cell transfection, protein extraction, western blot.