August 29, 2024 by University of Hawaii at Manoa Graphical abstract. Credit: Nucleic Acids Research (2024). DOI: 10.1093/nar/gkae534For decades, scientists have dreamed 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...
Tag: <span>Gene Therapy</span>
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New gene therapy approach shows promise for Duchenne muscular dystrophy
JULY 25, 2024 by Jackie Maupin, Indiana University School of Medicine Design and test of split intein constructs to assemble FL-dystrophin. Credit: Nature Communications (2024). DOI: 10.1038/s41467-024-50569-6Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments...
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Gene therapy discovery triggers hope for glaucoma patients
JULY 23, 2024 by Georgia Gowing, Macquarie University Modulation of tau expression in C57BL/6 mice retinas. Credit: Acta Neuropathologica Communications (2024). DOI: 10.1186/s40478-024-01804-0The protein tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells’ internal structures. These...
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Pancreatic Gene Therapy: A ‘One-and-Done’ GLP-1 Treatment?
MEDBRIEF Nancy A. Melville June 25, 2024 TOPLINE:An experimental pancreatic gene therapy given to a mouse model of obesity as a one-time, single-dose treatment showed improvements in body composition and fasting glucose comparable with those achieved with the glucagon-like peptide 1 (GLP-1) receptor agonist semaglutide, without the reversal of fat-loss and glycemia improvements that are...
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New gene delivery vehicle shows promise for human brain gene therapy
MAY 16, 2024 by Broad Institute of MIT and Harvard Credit: Unsplash/CC0 Public DomainIn an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene...
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Single-dose gene therapy may stop deadly brain disorders in their tracks
By Paul McClure A new single-dose genetic therapy has stopped the progression of two devastating neurodegenerative diseases in mice Depositphotos Researchers have developed a single-dose genetic therapy that can clear protein blockages that cause motor neurone disease, also called amyotrophic lateral sclerosis, and frontotemporal dementia, two incurable neurodegenerative diseases that eventually lead to death. In healthy...
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Not too late to repair: Gene therapy improves advanced heart failure in animal model
by Ana María Rodríguez, Baylor College of Medicine Credit: CC0 Public DomainHeart failure remains the leading cause of mortality in the U.S. During a heart attack blood stops flowing into the heart. Without oxygen, part of the heart muscle dies. The heart muscle does not regenerate; instead, it replaces dead tissue with a scar made of...
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Analysis shows gene therapy beneficial and likely cost-effective for patients with sickle cell disease
by American College of Physicians Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyA modeling study comparing the cost-effectiveness of gene therapy versus common care for patients with sickle cell disease found that gene therapy is beneficial in this patient population and likely cost-effective if the...
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Revolutionary gene therapy may offer new life for patients battling chronic sickle cell disease
by Robert McCoppin, Chicago Tribune Normal blood cells next to a sickle-blood cell, colored scanning electron microscope image. Credit: Wikipedia/Illustration from Anatomy & PhysiologyLyric Porter wanted her life back. Born with sickle cell disease, she had a fever at age 5 that scarred her lungs and required her to roll an oxygen tank to school....
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A novel switch to turn genes on/off on cue, a promising step toward safer gene therapy
Peer-Reviewed Publication BAYLOR COLLEGE OF MEDICINE Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important...