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Your search for “rare diseases” returned 212 results

UC San Diego Researchers Receive Funding to Pursue Novel Stem Cell-based Treatments

July 20, 2018

…of Medicine researchers investigating stem cell-based therapies for a rare genetic disorder that affects the heart and a chronic, progressive affliction of the lungs.

Domino Liver Transplant Treats Two Rare Diseases, World First

September 21, 2011

For the first time ever, a surgical team led by Alan Hemming, MD, has successfully performed a domino transplant using a liver with a rare genetic disorder called methylmalonic acidemia (MMA).

Rare Genetic Mutations Linked To Bipolar Disorder

December 21, 2011

An international team of scientists, led by researchers at the University of California, San Diego School of Medicine, reports that abnormal sequences of DNA known as rare copy number variants, or CNVs, appear to play a significant role in the risk for early onset bipolar disorder.

How a Virus Causes Chromosomal Breakage, Leading to Cancer

April 13, 2023

UC San Diego researchers describe for the first time how the Epstein-Barr virus exploits genomic weaknesses to cause cancer while reducing the body’s ability to suppress it.

Single Gene Mutation Linked to Diverse Neurological Disorders

October 9, 2013

…of Medicine, says a gene mutation that causes a rare but devastating neurological disorder known as Lesch-Nyhan syndrome appears to offer clues to the developmental and neuronal defects found in other, diverse neurological disorders like Alzheimer’s, Parkinson’s and Huntington’s diseases.

Center for AIDS Research Funding Renewed for an Old and On-Going Fight

April 16, 2018

The National Institute of Allergy and Infectious Diseases has awarded a five-year, $15 million grant to the San Diego Center for AIDS Research (CFAR) https://cfar.ucsd.edu at UC San Diego, renewing support that extends back to an original establishing grant in 1994—the height of the AIDS epidemic.

Artificial Intelligence Catalyzes Gene Activation Research and Uncovers Rare DNA Sequences

May 18, 2023

Biologists have used machine learning, a type of AI, to identify “synthetic extreme” DNA sequences with specifically designed functions in gene activation. They tested 50 million DNA sequences and found synthetic DNA sequences with activities that could be useful in biotechnology and medicine.

Cancer Drug and Antidepressants Provide Clues for Treating Fatal Brain-Eating Amoeba Infections

September 13, 2018

Researchers at Skaggs School of Pharmacy and Pharmaceutical Sciences at University of California San Diego have now identified three new molecular drug targets in Naegleria fowleri and a number of drugs that are able to inhibit the amoeba’s growth in a laboratory dish. Several of these drugs are already approved…

How Gene Therapy May Hold Key to Treating Life-Threatening Cardiac Disease

March 18, 2020

New study finds gene therapy improved cardiac, muscle and liver function in Danon disease mouse models.

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.

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