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Using CRISPR to Reverse Retinitis Pigmentosa and Restore Visual Function

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

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.

UC San Diego Launches Gene Therapy Initiative, Targeting Treatments for Rare Diseases

A $5 million gift from the Nancy and Geoffrey Stack Foundation has established the UC San Diego Gene Therapy Initiative to find novel therapies for children and adults with genetic diseases.

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

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.

UC San Diego Biologists Discover Genes That Repair Nerves After Injury

…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.

Genetic Testing Cost Effective for Newly Diagnosed GIST

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.

Gene Therapy Approach Shows Promise in Treating ALS

Researchers report that a gene therapy approach, developed at UC San Diego measurably delayed disease onset in humanized mouse and rat models of familial ALS, an inherited form of the disease that runs in families.

Gene Therapy Finds New Life in an Old Home

An international symposium November 17-18, 2022 at UC San Diego will discuss current clinical trials using gene therapy, here and abroad.

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

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.

Personalizing Precision Medicine with Combination Therapies Improves Outcomes in Cancer

University of California San Diego School of Medicine researchers found that treating patients with personalized precision medicine that combined therapies to target multiple alterations improved outcomes in patients with therapy resistant cancers.