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

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

Author: UCTV March 11, 2026 Duration: 20:15
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. Series: "Stem Cell Channel" [Health and Medicine] [Show ID: 41166]

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Author: Language: en-us Episodes: 100

University of California Video Podcasts (Video)
Podcast Episodes
CARTA: The Idea Organ - Questions Answers and Closing Remarks [not-audio_url] [/not-audio_url]

Duration: 1:03:38
Humans live in a world of ideas—born in the brain, shared through language, accumulated in culture across generations, and made reality. From the first flaked stone tools to the building of shelters, from figurative and…
Where Are We Now? Bias in Health AI [not-audio_url] [/not-audio_url]

Duration: 35:15
Bias in health AI can shape who gets care, how fairly risk is measured, and whether automation helps or harms patients. Karandeep Singh, M.D., M.M.S.C. explains that predictive AI can reflect historical, representation,…
Is This Your Only Life? [not-audio_url] [/not-audio_url]

Duration: 1:20:13
Embodiment affects how we understand personhood, moral status, and whether this life is our only life. Mark Johnston, Henry Putnam University Professor, Princeton University, explains how competing theories of mind and m…
CARTA: The Costs of Big Brains with Alex Pollen [not-audio_url] [/not-audio_url]

Duration: 17:00
Human brain expansion is often discussed in terms of the genetic and molecular innovations that drove uniquely human cognitive abilities. Yet evolution is fundamentally a process of tradeoffs. Disproportionate expansion…
Artificial Intelligence in (AI-Driven) Healthcare [not-audio_url] [/not-audio_url]

Duration: 39:36
AI in healthcare raises urgent questions about bias, privacy, and power. Safiya U. Noble, Ph.D., examines how AI systems can reproduce social and racial inequities when they rely on incomplete data, hidden assumptions, a…
The Impact of AI on Business [not-audio_url] [/not-audio_url]

Duration: 44:02
Artificial intelligence is changing how businesses use data, make decisions, and organize work. UC San Diego Rady School of Management's Thomas Beyer looks at that evolution from the early push to collect as much data as…