Development of a Multivalent Gene Therapy to Correct Cryptic Splicing in ALS скачать в хорошем качестве

Development of a Multivalent Gene Therapy to Correct Cryptic Splicing in ALS

RNA binding proteins help cells control how genetic information becomes working proteins, and Gene Yeo, Ph.D., M.B.A., at UC San Diego investigates how their disruption contributes to neurodegenerative disease. Yeo focuses on ALS, a severe motor neuron disease in which the RNA binding protein TDP-43 moves from the nucleus to the cytoplasm, loses normal RNA processing functions, and triggers cryptic exons that damage key neuronal genes, including one linked to motor neuropathy. His group maps these RNA changes and develops small nuclear RNA guides packaged in AAV vectors to block harmful splice sites and restore healthy RNA and protein levels. In cell cultures and a humanized mouse model, this strategy improves axon growth and supports the idea that multiplexed RNA-targeted therapies could correct multiple disease pathways at once. [Show ID: 41166] Donate to UCTV to support informative & inspiring programming: https://www.uctv.tv/donate Please Note: Knowledge about health and medicine is constantly evolving. This information may become out of date. More videos from: Sanford Stem Cell Institute Symposium 2025 (https://www.uctv.tv/stem-cell/sanford...) More from: Stem Cell Channel (https://www.uctv.tv/stem-cell) Explore More Health & Medicine on UCTV (https://www.uctv.tv/health) UCTV features the latest in health and medicine from University of California medical schools. Find the information you need on cancer, transplantation, obesity, disease and much more. UCTV is the broadcast and online media platform of the University of California, featuring programming from its ten campuses, three national labs and affiliated research institutions. UCTV explores a broad spectrum of subjects for a general audience, including science, health and medicine, public affairs, humanities, arts and music, business, education, and agriculture. Launched in January 2000, UCTV embraces the core missions of the University of California -- teaching, research, and public service – by providing quality, in-depth television far beyond the campus borders to inquisitive viewers around the world. (https://www.uctv.tv)