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Your search for “gene therapy” returned 715 results

“Soft” CRISPR May Offer a New Fix for Genetic Defects

July 1, 2022

Scientists have developed a CRISPR-based technology that could offer a safer approach to correcting genetic defects. The new “soft” CRISPR system makes use of natural DNA repair machinery, providing a foundation for novel gene therapy strategies with the potential to cure genetic diseases.

Brain Cancer Cells Hide While Drugs Seek

December 5, 2013

…of Medicine, has found that brain cancer cells resist therapy by dialing down the gene mutation targeted by drugs, then re-amplify that growth-promoting mutation after therapy has stopped.

Even DNA that Doesn’t Encode Genes Can Drive Cancer

April 2, 2018

The vast majority of genetic mutations associated with cancer occur in non-coding regions of the genome, yet it’s unclear how they may influence tumor development or growth. Researchers at University of California San Diego School of Medicine and Moores Cancer Center identified nearly 200 mutations in non-coding DNA that play…

Gene Therapy Reverses Effects of Autism-Linked Mutation in Brain Organoids

May 2, 2022

UC San Diego scientists use lab-grown human brain tissue to identify neural abnormalities in Pitt-Hopkins Syndrome and show gene therapy tools can rescue neural structure and function.

Proposed Drug May Reverse Huntington’s Disease Symptoms

June 20, 2012

With a single drug treatment, researchers at the Ludwig Institute for Cancer Research at the University of California, San Diego School of Medicine can silence the mutated gene responsible for Huntington’s disease, slowing and partially reversing progression of the fatal neurodegenerative disorder in animal models.

AI Predicts How Patients with Viral Infections, Including COVID-19, Will Fare

June 11, 2021

UC San Diego School of Medicine researchers discovered gene expression patterns associated with pandemic viral infections, providing a map to help define patients’ immune responses, measure disease severity, predict outcomes and test therapies — for current and future pandemics.

Boosting A Natural Protection Against Alzheimer’s Disease

March 12, 2015

…California, San Diego School of Medicine have identified a gene variant that may be used to predict people most likely to respond to an investigational therapy under development for Alzheimer’s disease (AD). The study, published March 12 in Cell Stem Cell, is based on experiments with cultured neurons derived from…

Gene Therapy in Alzheimer’s Disease Mouse Model Preserves Learning and Memory

May 4, 2021

Researchers at UC San Diego have used gene therapy to prevent learning and memory loss in a mouse model of Alzheimer’s disease, a key step toward eventually testing the approach in humans with the neurodegenerative disease.

Genetic Adaptation Keeps Ethiopians Heart-Healthy Despite High Altitudes

August 3, 2015

…and hypoxia is the endothelin receptor type B (EDNRB) gene. Researchers at University of California, San Diego School of Medicine now find that mice with lower-than-normal levels of EDNRB protein are remarkably tolerant to hypoxia.

Degenerating Neurons Respond to Gene Therapy Treatment for Alzheimer’s Disease

August 27, 2015

Degenerating neurons in patients with Alzheimer’s disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, report researchers at University of California, San Diego School of Medicine in the current issue of JAMA Neurology.

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