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Your search for “gene therapy” returned 741 results

Using CRISPR to Reverse Retinitis Pigmentosa and Restore Visual Function

April 21, 2017

Using the gene-editing tool CRISPR/Cas9, researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China, have reprogrammed mutated rod photoreceptors to become functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of…

“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.

New Resource Makes Gene Editing Technology Even More User-Friendly

July 17, 2015

…to accompany the powerful gene editing tool called CRISPR/Cas9, which has been widely adopted to make precise, targeted changes in DNA. This breakthrough has the potential to facilitate new discoveries in gene therapies and basic genetics research. The research was published in the July 13 issue of Nature Methods.

Alternative Proteins Encoded by Same Gene Have Widely Divergent Functions

February 11, 2016

A single human gene can produce many different proteins. In the first large-scale study of its kind, researchers at University of California, San Diego School of Medicine, Dana-Farber Cancer Institute and McGill University report that most of these sibling proteins encoded by the same gene — known as protein isoforms…

Researchers Discover Specific Tumor Environment That Triggers Cells to Metastasize

November 21, 2017

…found that they turn on a specific set of genes and begin to form structures that resemble blood vessels.

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.

New Research Helps Explain the Genetic Basis of Why We Look the Way We Do

November 10, 2021

Comparing features of a common laboratory fruit fly with its rarer cousin collected from Hawai’i Volcanoes National Park, UC San Diego researchers used CRISPR technology to uncover clues about how high-level control genes called Hox genes shape our appearance.

Genetic Testing Cost Effective for Newly Diagnosed GIST

September 28, 2020

UC San Diego School of Medicine researchers reported that genetic testing is cost-effective and beneficial for newly diagnosed patients with metastatic gastrointestinal stromal tumors (GIST), a rare type of cancer.

Study Identifies Protein Essential for Normal Heart Function

June 17, 2013

A study by researchers at Skaggs School of Pharmacy and Pharmaceutical Sciences and the Department of Pharmacology at the University of California, San Diego, shows that a protein called MCL-1, which promotes cell survival, is essential for normal heart function.

Phage Therapy Shows Promise Beyond Treating Infections

November 14, 2019

…almost killed him. Phage Therapy Shows Promise Beyond Treating Infections For first time, researchers use bacteriophages in mice to treat a condition not considered a classic bacterial infection: alcoholic liver disease. Bacteriophages are viruses that specifically destroy bacteria. In the early 20th century, researchers experimented with phages as a potential…

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