Health and Medicine (Audio)

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]

As part of the 2026 UCSF Patient Conference on Prostate Cancer, Dr. Steven Seyedin discusses radiation therapy. Series: "Prostate Cancer Patient Conference" [Health and Medicine] [Show ID: 41558]

Pre-cancer and cancer can begin when stressed blood-forming stem cells lose their normal controls. Catriona Jamieson, M.D., Ph.D., UC San Diego, explains how inflammation-linked editing enzymes, repetitive elements in the genome, and stem cell stress shape the progression from myeloproliferative neoplasms to acute myeloid leukemia. Jamison examines how spaceflight accelerates stem cell aging, how some astronauts mobilize a resilient regenerative stem cell population, and how tumor organoids in space help reveal drug responses by activating the enzyme ADAR1. This work helps explain how cancer starts, why it can return, and how space-based research may speed the development of therapies that stop malignant stem cells before disease advances. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 41473]

As part of the 2026 UCSF Patient Conference on Prostate Cancer, Dr. Spencer Behr discusses PSMA PET and functional imaging. Series: "Prostate Cancer Patient Conference" [Health and Medicine] [Show ID: 41557]

As part of the 2026 UCSF Patient Conference on Prostate Cancer, Dr. Jonathan Chou discusses targeted therapies. Series: "Prostate Cancer Patient Conference" [Health and Medicine] [Show ID: 41563]