Targeted inhibitor of mutated KRAS gene shows promise in lung, bowel, other solid tumors

A novel agent that targets a mutated form of the KRAS gene—the most commonly altered oncogene in human cancers and one long considered “undruggable”—shrank tumors in most patients in a clinical trial with manageable side effects, researchers reported today at the 32nd EORTC-NCI-AACR Symposium on Molecular Targets and Therapeutic, which is taking place online. The KRYSTAL-1 (NCT03785249) phase I/II trial […]

Read more

Study reveals restoration of retinal and visual function following gene therapy

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease. Published today in Nature Biomedical Engineering, the paper, titled, “Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing,” illustrates the use of a new […]

Read more

Inflammatory gene provides clue to obesity risk

A gene that helps to control inflammation increases the risk of obesity and could be turned off in mice to stop weight gain, a study from The University of Queensland has found. UQ Institute for Molecular Bioscience researcher Dr. Denuja Karunakaran said she was determined to unravel the links between inflammation and obesity that went deeper than excessive eating or […]

Read more

Using gene therapy to treat chronic traumatic encephalopathy

A new study shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study, which demonstrated the effectiveness of direct delivery of gene therapy into the brain of a mouse model of CTE, is published in Human Gene Therapy. Ronald Crystal and colleagues from Weill Cornell Medical College, New York, NY, coauthored […]

Read more

New technology allows control of gene therapy doses

Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing. The feat, reported in the scientific journal Nature Biotechnology, offers gene therapy designers what may be the first viable technique for adjusting the activity levels of their therapeutic genes. The lack of such a basic […]

Read more

Injection of virus-delivered gene silencer blocks ALS degeneration, saves motor function

Writing in Nature Medicine, an international team headed by researchers at University of California San Diego School of Medicine describe a new way to effectively deliver a gene-silencing vector to adult amyotrophic lateral sclerosis (ALS) mice, resulting in long-term suppression of the degenerative motor neuron disorder if treatment vector is delivered prior to disease onset, and blockage of disease progression […]

Read more

Study identifies role of specific gene in hardening of blood vessel walls

Arterial wall calcification is the buildup of calcium in the blood vessel walls, which can often be a predictor of serious cardiovascular events like heart attacks and strokes. Scientists have long pointed to family history as one possible cause for this hardening of the arteries, but a new study published in Nature Genetics implicates a specific gene—HDAC9—in the calcification of […]

Read more

Researchers create new viral vector for improved gene therapy in sickle cell disease

Researchers at the National Institutes of Health have developed a new and improved viral vector—a virus-based vehicle that delivers therapeutic genes—for use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently […]

Read more
1 2 3