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Genetics (Audio)University of California Television Author: UCTV
University of California faculty present the latest findings in genetic research and discoveries. Language: en-us Genres: Health & Fitness, Medicine, Science Contact email: Get it Feed URL: Get it iTunes ID: Get it |
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Gene Therapies and Rare Disease - Medicine Informing Novel Discoveries (MIND)
Friday, 12 June, 2026
Rare disease research is creating new paths for diagnosis, treatment, and broader medical discovery. Gene therapy can repair or replace faulty genes, and work on cystinosis has led to a stem cell platform now being applied to Danon disease, Sanfilippo syndrome C, Friedreich’s ataxia, and Alzheimer’s research. Funding programs support gene therapy, clinical trials, and new platform approaches for rare diseases. CAR-T cell research is also advancing treatment possibilities for pediatric brain tumors, including early results in children with DIPG and diffuse midline glioma. A patient advocate shares her daughter’s diagnostic odyssey and treatment for TUBB4A leukodystrophy. Together, these stories show why rare disease research matters beyond rarity. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 41402]
