16 March 2025
Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
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One gene variant is poised to cure a devastating inherited disease
For more than 15 years, a pulmonologist has tried to understand the random path of devastation that a rare genetic condition carves through the families it affects.
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Gene therapy for rare epilepsy shows promise in mice
Recent research takes aim at the a variant in gene SCN1B, which causes a severe form of developmental epileptic encephalopathy.
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Gene modification could improve cancer treatment's success rate
A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 gene helps CAR-T cells fight cancer more effectively. These findings could improve immunotherapy for patients with aggressive cancers such as leukemia.
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Histone modification important for correct blood cell formation
Molecular biologists decode the epigenetic silencing of problematic retroviral gene sequences.
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New gene therapy reverses heart failure in large animal model
In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves survival.
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Use of 'genetic scissors' carries risks
The CRISPR tool is capable of repairing the genetic defect responsible for the immune disease chronic granulomatous disease. However, researchers have now shown that there is a risk of inadvertently introducing other defects.
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Researchers discover underlying mechanisms that make CRISPR an effective gene editing tool
Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing function. These preclinical findings could lead to improved designs for CRISPR-based gene editing.
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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 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.
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Effective new gene therapy to treat multiple sulfatase deficiency
Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, skin, and skeleton with no currently approved treatments. The findings demonstrated several improvements in outcomes, paving the way for future translation into clinical trials.
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New gene therapy offers promise for treating glaucoma -- as well as AMD
Scientists developed a highly promising gene therapy to treat glaucoma -- a debilitating eye condition that can lead to complete vision loss, and which affects around 80 million people worldwide. The team previously showed their therapy offered promise in treating dry age-related macular degeneration (AMD).
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Gene therapy gets a turbo boost
For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes. Researchers have now made a significant breakthrough in gene editing technology that could revolutionize how we treat genetic diseases.
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Scientists discover new code governing gene activity
A newly discovered code within DNA -- coined 'spatial grammar' -- holds a key to understanding how gene activity is encoded in the human genome. This breakthrough finding revealed a long-postulated hidden spatial grammar embedded in DNA. The research could reshape scientists' understanding of gene regulation and how genetic variations may influence gene expression in development or disease.
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Noninvasive measurement of gene expression at target locations in the brain
Bioengineers have developed a noninvasive tool to measure gene expression and gene therapy delivery in specific brain regions using ultrasound.
