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Your search for “Gene Therapy” returned 776 results

Editing Genes One by One Throughout Colorectal Cancer Cell Genome Uncovers New Drug Targets

September 27, 2017

…mutations in the KRAS gene are among the most deadly. For decades, researchers have tried unsuccessfully to directly target mutant KRAS proteins as a means to treat tumors. Instead of targeting mutant KRAS itself, researchers at University of California San Diego School of Medicine are now looking for other genes…

Researchers Find Fertility Genes Required for Sperm Stem Cells

September 27, 2016

…of transcription factors — regulatory proteins that activate some genes and inactivate others — drive the development of stem cells in the testes in mice. The investigators also linked RHOX gene mutations to male infertility in humans.

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…

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 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.

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…

First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer’s Disease

February 18, 2021

UC San Diego researchers have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer’s disease or Mild Cognitive Impairment, a condition that often precedes full-blown dementia.

Large Study Reveals PTSD Has Strong Genetic Component Like Other Psychiatric Disorders

October 8, 2019

In the largest and most diverse genetic study of PTSD to date, scientists from UC San Diego School of Medicine and more than 130 institutions in the Psychiatric Genomics Consortium found that genetics accounts for five to 20 percent of the variability in PTSD risk following a traumatic event.

New Genetic Analysis Tool Tracks Risks Tied to CRISPR Edits

March 26, 2024

While CRISPR has shown immense promise as a next-generation therapeutic tool, the gene editing technology’s edits are still imperfect. Researchers have developed a new genetic system to test and analyze CRISPR-based DNA repair and related risks from unintended but harmful “bystander” edits.

UC San Diego Biologists Discover Genes That Repair Nerves After Injury

September 21, 2011

…of California, San Diego have identified more than 70 genes that play a role in regenerating nerves after injury, providing biomedical researchers with a valuable set of genetic leads for use in developing therapies to repair spinal cord injuries and other common kinds of nerve damage such as stroke.

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