by Flora Hui and Guei-Sheung (Rick) Liu, The Conversation Credit: Pixabay/CC0 Public Domain The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA editing. Its aim is to treat an eye disease called wet age-related macular degeneration that causes vision loss in millions of older people worldwide. This trial marks a new...
Tag: <span>Gene Therapy</span>
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Discovery enables effective use of gene therapy for muscular dystrophies and other large-gene diseases
by University of Rochester Medical Center Credit: Pixabay/CC0 Public Domain Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular dystrophies are often extremely large, and current delivery methods can’t courier such substantial genetic loads into the body....
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Gene therapy protects against motor neuron disease in rats
by Will Cushman, University of Wisconsin-Madison A section of brain tissue from a rat that received a gene therapy targeting the Trk-fused gene. A mutation in that gene is implicated in the development of certain motor neuron diseases. Green arrows highlight neurons expressing elevated levels of the gene following the therapy. Red arrows indicate other nerve...
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Is gene therapy the next big step in vision loss treatment?
Diseases that affect the retina, the light-sensitive layer at the back of the eye, are a significant cause of visual impairment and blindness. Gene therapy holds promise for treating some of these conditions, and current research advances may soon shift the therapeutic landscape for eye health. However, many obstacles remain in place, as this Special...
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Improving the recipe for gene therapy: New treatment for Gaucher disease shows promise
MRI brain scans of mice with Gaucher disease Type III. The scan of a mouse that received the new treatment (left) shows a significant reduction in tissue inflammation (blue) compared to an untreated mouse (right). Credit: Weizmann Institute of Science Gregory and Deborah Macres from California lost their son, Gregory, in 1997, when he was...
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Gene therapy that converts omega-6 to omega-3 fatty acids in the body could combat effects of childhood obesity
Credit: Unsplash/CC0 Public Domain According to the Centers for Disease Control, nearly 20% of children and teens are considered obese. Research shows it can have a dramatic impact on a variety of health conditions, including arthritis, heart conditions and other metabolic problems, and the American Academy of Pediatrics now recommends early and intensive treatment to...
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Magnetically regulated gene therapy tech offers precise brain-circuit control
A gene therapy allows precise magnetic field control of specific brain circuits without implanted devices. The image shows restricted mRNA expression of the genetically encoded magnetic sensor (red) in dopaminergic neurons type 2 (green) in the mouse striatum that regulate initiation of movement. Dopaminergic neurons type 1 (cyan) and cell nuclei shown with dapi staining...
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Single-dose gene therapy is potentially life-changing for adults with hemophilia B
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71% after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published in the New England Journal of Medicine by researchers from the University of Pennsylvania Perelman School of Medicine and a...
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100x Improvement in Sight Seen After Gene Therapy Trial
SCIENCE & TECHNOLOGY NEWS The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it. Some patients even experienced a 10,000-fold improvement in their vision after receiving the highest...
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100-fold improvement in sight seen after gene therapy trial
September 5, 2024 by Perelman School of Medicine at the University of Pennsylvania Credit: Unsplash/CC0 Public Domain The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it. Some...