Genetic Mutations of Appendix Cancer Identified, May Impact Treatment
August 8, 2018
…appendiceal tumors — the largest such study of this disease to date — to compare mutations present in both cancer types.
Your search for “Rare Disease” returned 219 results
Dysfunction in Neuronal Transport Mechanism Linked to Alzheimer’s Disease
October 12, 2016
…underappreciated role in promoting the heritable form of Alzheimer’s disease (AD), but also one that might be remedied with existing therapeutic enzyme inhibitors.
Stem Cell-derived “Mini-brains” Reveal Potential Drug Treatment for Rare Disorder
September 8, 2015
Using “mini-brains” built with induced pluripotent stem cells derived from patients with a rare, but devastating, neurological disorder, researchers at University of California, San Diego School of Medicine say they have identified a drug candidate that appears to “rescue” dysfunctional cells by suppressing a critical genetic alteration.
Researchers Create Model of Anorexia Nervosa Using Stem Cells
March 14, 2017
An international research team, led by scientists at University of California San Diego School of Medicine, has created the first cellular model of anorexia nervosa (AN), reprogramming induced pluripotent stem cells (iPSCs) derived from adolescent females with the eating disorder.
Selective, Toxin-Bearing Antibodies Could Help Treat Liver Fibrosis
July 12, 2021
UC San Diego researchers discovered that immunotoxins targeting the protein mesothelin prevent liver cells from producing collagen, a precursor to fibrosis and cirrhosis, in mouse models of human disease.
Gene Therapy Finds New Life in an Old Home
November 10, 2022
An international symposium November 17-18, 2022 at UC San Diego will discuss current clinical trials using gene therapy, here and abroad.
New wearable sensor tracks Vitamin C levels in sweat
May 18, 2020
A team at the University of California San Diego has developed a wearable, non invasive Vitamin C sensor that could provide a new, highly personalized option for users to track their daily nutritional intake and dietary adherence. The study was published in the May 18, 2020 issue of ACS Sensors.
Smartly Done
May 18, 2023
Across disciplines and specialties, from cells to systems, artificial intelligence is on its way to altering how we understand and treat disease.
Promising Clinical Trial Results for Drug for Rare Disease in Which Patients Can’t Eat Fat
August 7, 2019
In a Phase III clinical trial, the drug volanesorsen significantly reduced blood fat (triglyceride) levels in participants with a rare disease called familial chylomicronemia syndrome; finding could also help inform better prevention methods and treatments for many types of heart disease.
CAR Trials Drive Leukemia and Lymphoma Treatment in New Direction
February 23, 2016
Cancer immunology is based upon boosting the body’s own immune system to vanquish malignancies. It is among the fastest growing areas of oncology research. Researchers at UC San Diego Moores Cancer Center have launched three clinical trials to test the safety and efficacy of a novel cellular-immunotherapy that uses modified…
