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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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.
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New gene therapy approach shows promise for Duchenne muscular dystrophy
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 for people with Duchenne muscular dystrophy (DMD).
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New gene therapy for muscular dystrophy offers hope
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles. The research focuses on delivering a series of protein packets inside shuttle vectors to replace the defective DMD gene within the muscles.The gene for dystrophin is one of the largest in the human genome, and is difficult to fit inside a delivery shuttle. Instead of one shuttle, the new gene therapy uses a series of shuttles which take parts of the therapeutic gene inside the muscles, along with embedded instructions to begin assembling the fragments once within the body. The assembled genetic code will then start producing dystrophin, the protein lacking in patients with muscular dystrophy.
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With gene editing, mice with a form of inherited deafness can hear again
Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled the animals to regain hearing. The approach may eventually lead to potential treatments for inherited hearing loss in people.
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Novel genome editing approach restores hearing in adult preclinical models with genetic deafness
Researchers restored hearing in preclinical mouse models with a specific form of inherited deafness called DFNA50 caused by mutations in microRNA, by using a novel in vivo CRISPR genome editing approach. Since mouse and human microRNAs have identical sequences, the researchers hope this work can one day be translated into applications for humans.
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Researchers discover potential mole reversal therapy in rare condition
Researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition.
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Promising gene therapy for FOXG1 syndrome
A viral gene therapy has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental disorder.
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Improved prime editing system makes gene-sized edits in human cells at therapeutic levels
Scientists have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for therapeutic applications. The advance could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene. Using this new approach, they would insert a healthy copy of the gene at its native location in the genome, rather than having to create a different gene therapy to correct each mutation using other gene-editing approaches that make smaller edits.
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New, modified CRISPR protein can fit inside virus used for gene therapy
Researchers have developed a novel version of a key CRISPR gene-editing protein that shows efficient editing activity and is small enough to be packaged within a non-pathogenic virus that can deliver it to target cells.
