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Timothy syndrome (TS) is a severe, multisystem disorder characterized by autism, epilepsy, long-QT syndrome and other neuropsychiatric conditions1. TS type 1 (TS1) is caused by a gain-of-function variant in the alternatively spliced and developmentally enriched CACNA1C exon 8A, as opposed to its counterpart exon 8. We previously uncovered several phenotypes in neurons derived from patients with TS1, including delayed channel inactivation, prolonged depolarization-induced calcium rise, impaired interneuron migration, activity-dependent dendrite retraction and an unanticipated persistent expression of exon 8A2–6. We reasoned that switching CACNA1C exon utilization from 8A to 8 would represent a potential therapeutic strategy. Here we developed antisense oligonucleotides (ASOs) to effectively decrease the inclusion of exon 8A in human cells both in vitro and, following transplantation, in vivo. We discovered that the ASO-mediated switch from exon ....
Global Biochemical Market Forecast 2021-2031 openpr.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from openpr.com Daily Mail and Mail on Sunday newspapers.
Proteinase K Market Size, Share, Revenue, Trends Analysis And Drivers For 2023-2032 einnews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from einnews.com Daily Mail and Mail on Sunday newspapers.
Worthington Biochemical (WBC) has recently unveiled the latest edition of its comprehensive Enzyme and Biochemical Catalogue, now available in both print and digital formats. Click to read more. ....
Collagenase is medically used to breakdown the tissue, cancers cell clusters and other large grouped cells and tissue that are considered harmful to the body. In several biochemical processes, primary cell extraction and tissue disintegration techniques, crude collagenases are frequently utilized.Access the sample r. ....