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 […]

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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 […]

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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 […]

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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 […]

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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 […]

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Gene protective against fruit fly heat-induced seizures may explain some human seizures

Researchers identified a gene in fruit flies that helps prevent the hyperexcitability of specific neurons that trigger seizures. In humans, mutations in the gene may be linked to seizures associated with Long QT Syndrome. A research team led by Yehuda Ben-Shahar of Washington University in St. Louis report these findings in a paper published 8th August in PLOS Genetics. The […]

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

In a paper published in the July 31 issue of Science Translational Medicine, researchers at Fred Hutchinson Cancer Research Center 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. It’s the first time that scientists have specifically edited the genetic makeup of a specialized subset […]

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Proposed gene therapy for a heart arrhythmia, based on models made from patient cells

Researchers at Boston Children’s Hospital report creating the first human tissue model of an inherited heart arrhythmia, replicating two patients’ abnormal heart rhythms in a dish, and then suppressing the arrhythmia with gene therapy in a mouse model. Their work, published in two papers in the July 30 print issue of the journal Circulation, opens the possibility of developing single-dose […]

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