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Science Daily Gene Therapy News

ScienceDaily is best known for showcasing the top science news stories from the world’s leading universities and research organizations. These current stories on gene therapy are selected from among dozens of press releases and other materials submitted to ScienceDaily every day.


  • Unique case of disease resistance reveals possible Alzheimer's treatment

    Nov 4, 2019 | 17:28 pm

    Defying the odds, an individual at high risk for early-onset Alzheimer's disease remained dementia-free for many years beyond what was anticipated. A new study led researchers to suggest that a gene variant may be the key, perhaps providing a new direction toward developing a treatment.

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  • New gene therapy for epilepsy provides on-demand release of endogenous substance

    Oct 30, 2019 | 16:00 pm

    Scientists have developed a new therapeutic concept for the treatment of temporal lobe epilepsy. It represents a gene therapy capable of suppressing seizures at their site of origin on demand. Having been shown to be effective in an animal model, the new method will now be optimized for clinical use.

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  • Discovery in monkeys could lead to treatment for blindness-causing syndrome

    Oct 25, 2019 | 20:53 pm

    A genetic mutation that leads to a rare, but devastating blindness-causing condition called Bardet-Biedl Syndrome has been discovered in monkeys for the first time. The finding offers a promising way to develop gene and cell therapies that could treat people with the condition, which leads to vision loss, kidney disfunction, extra fingers or toes, and other symptoms.

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  • Deleting a liver enzyme lowers the health risk of sweet treats (at least in mice)

    Oct 24, 2019 | 16:58 pm

    Hepatic insulin resistance, caused by diets high in sugar and fat, can lead to type 2 diabetes. Researchers found that the Elovl6 gene plays a key role in hepatic insulin resistance. Deleting Elov6 in liver cells causes a rise in a specific ceramide lipid that protects mice from hepatic insulin resistance due to excessive dietary sugar. The findings could help efforts to find a targeted treatment for the condition.

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  • When added to gene therapy, plant-based compound may enable faster, more effective treatments

    Oct 17, 2019 | 22:22 pm

    Today's standard process for administering gene therapy is expensive and time-consuming -- a result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem. Scientists believe they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.

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  • In-office gene therapy for wet age-related macular degeneration is coming

    Oct 11, 2019 | 13:47 pm

    Gene therapy is showing promise for one of the most common causes of blindness.

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

    Oct 2, 2019 | 16:28 pm

    Researchers 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 used, and it had a carrying capacity of up to six times higher, the researchers report.

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  • Researchers advance search for safer, easier way to deliver vision-saving gene therapy

    Sep 30, 2019 | 14:23 pm

    In experiments with rats, pigs and monkeys, researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a new, more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration (AMD), and it could potentially replace defective genes in patients with inherited retinal disease.

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  • Researchers developing new 'DNA stitch' to treat muscular dystrophy

    Sep 25, 2019 | 19:36 pm

    A new therapeutic being tested is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD). The treatment -- a cocktail of DNA-like molecules -- results in dramatic regrowth of a protein called dystrophin, which acts as a support beam to keep muscles strong. The protein is virtually absent in those with DMD.

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  • Non-viral gene therapy to speed up cancer research

    Sep 24, 2019 | 16:41 pm

    A new treatment method promises to speed up gene therapy research and could bring new, patient friendly cancer treatments to market faster.

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  • World's first gene therapy for glycogen storage disease produces remarkable results

    Sep 20, 2019 | 18:46 pm

    The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. A clinical trial originally set out to simply test the safety and dosage of the gene therapy for three patients with GSD Type Ia. The dramatic improvement in their lives was unexpected.

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  • New protocol to improve gene therapy tool production

    Sep 19, 2019 | 18:25 pm

    A method to create a faster and lower cost alternative for a gene therapy tool.

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  • Tiny bubbles in our body could fight cancer better than chemo

    Sep 13, 2019 | 17:13 pm

    Healthy cells in our body release nano-sized bubbles that transfer genetic material such as DNA and RNA to other cells. It's your DNA that stores the important information necessary for RNA to produce proteins and make sure they act accordingly. These bubbly extracellular vesicles could become mini treatment transporters, carrying a combination of therapeutic drugs and genes that target cancer cells and kill them, according to new research from Michigan State University and Stanford University.

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  • Success of gene therapy for a form of inherited blindness depends on timing

    Sep 9, 2019 | 23:07 pm

    An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients' sight. But new research on the blinding condition but new research underscores the importance of further investigation to halt the progression of the disorder.

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  • Chronic cocaine use modifies gene expression

    Sep 2, 2019 | 19:53 pm

    Chronic cocaine use changes gene expression in the hippocampus, according to new research in mice.

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  • Gene therapy reduces obesity and reverses type 2 diabetes in mice

    Aug 29, 2019 | 21:07 pm

    The obesity epidemic affects nearly half a billion people worldwide, many of them children. Obesity-related diseases including heart disease, stroke, type 2 diabetes, and cancer are a leading cause of preventable death. Researchers have now developed a gene therapy that specifically reduces fat tissue and reverses obesity-related metabolic disease in obese mice.

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  • Promising approach to reducing plaque in arteries

    Aug 26, 2019 | 18:19 pm

    In a new study, investigators have revealed previously unknown factors that contribute to the hardening of arteries and plaque growth, which cause heart disease. Their insight is the basis for a promising therapeutic approach to halt and potentially reverse plaque buildup and the progression of disease, the researchers said.

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  • Mutations linked to familial pancreatic cancer

    Aug 15, 2019 | 18:51 pm

    A new study finds genetic mutations associated with hereditary forms of pancreatic cancer and mechanism by which these mutations may contribute to the development of tumors.

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  • Inherited pancreatic cancer risk mutation identified

    Aug 12, 2019 | 19:08 pm

    The discovery of the previously unknown mutation could lead to routine testing of individuals with a strong family history of pancreatic cancer to determine if they carry the mutation, occurring in the gene known as RABL3.

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  • Targeting a blood stem cell subset shows lasting, therapeutically relevant gene editing

    Jul 31, 2019 | 20:58 pm

    Researchers have used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several blood disorders, including sickle cell disease and beta-thalassemia.

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