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Transcripts For KQED Charlie Rose 20121126

Captioning sponsored by Rose Communications from our studios in new york city, this is charlie rose. Tonight we continue to exploring our brain with the conversation about pain. Pain serves a very important function for us to survive, it teaches us what to avoid and lets us know when to seek medical help. At the same time, though it can create tremendous suffering. St. Augustine once said the greatest evil is physical pain, 100 million americans live with it every day would yo would wouo doubt agree, pain knows no boundaries, regardless of age and race, beyond the physical symptoms the experience of chronic pain often leads to feelings of isolation and hopelessness. Laura klein had been living with pain since a knee injury in 2008 and joins me this evening to speak about her experiences and Incredible Group of scientists are also here to discuss how we perceive and process pain, david bar stiewk of Childrens Hospital and david julius of the university of california, san francisco, alla

Transcripts For KQED Charlie Rose 20121124

Captioning sponsored by Rose Communications from our studios in new york city, this is charlie rose. Tonight we continue to exploring our brain with the conversation about pain. Pain serves a very important function for us to survive, it teaches us what to avoid and lets us know when to seek medical help. At the same time, though it can create tremendous suffering. St. Augustine once said the greatest evil is physical pain, 100 million americans live with it every day would yo would wouo doubt agree, pain knows no boundaries, regardless of age and race, beyond the physical symptoms the experience of chronic pain often leads to feelings of isolation and hopelessness. Laura klein had been living with pain since a knee injury in 2008 and joins me this evening to speak about her experiences and Incredible Group of scientists are also here to discuss how we perceive and process pain, david bar stiewk of Childrens Hospital and david julius of the university of california, san francisco, alla

Antisense oligonucleotide therapeutic approach for Timothy syndrome

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 

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