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Science Daily Gene Therapy News

ScienceDaily is best known for showcasing the top science news stories from the world’s leading universities and research organizations. These current stories on gene therapy are selected from among dozens of press releases and other materials submitted to ScienceDaily every day.


  • Scientists take major step toward Angelman Syndrome gene therapy

    Oct 21, 2020 | 17:23 pm

    Babies born with a faulty maternal copy of the UBE3A gene will develop Angelman syndrome, a severe neurodevelopmental disorder with no cure and limited treatments. Now, for the first time, scientists show that gene editing and gene therapy techniques can be used to restore UBE3A in human neuron cultures and treat deficits in an animal model of Angelman syndrome.

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  • Restoration of retinal and visual function following gene therapy

    Oct 19, 2020 | 21:59 pm

    A breakthrough study results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

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  • Advanced prostate cancer has an unexpected weakness that can be targeted by drugs

    Oct 7, 2020 | 18:30 pm

    Researchers reported that the SUCLA2 gene is frequently involved in the deletion of the tumor suppressor gene RB1 in advanced prostate cancer. RB1 deletion makes cells resistant to hormone therapy but SUCLA2 deletion induces a metabolic weakness. The study showed that thymoquinone selectively killed SUCLA2-deficient prostate cancer cells in vitro and in vivo. The findings highlight a vulnerability of advanced prostate cancer cells that can be targeted by drugs.

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  • Gene therapy: Novel targets for congenital blindness

    Sep 2, 2020 | 15:51 pm

    Retinitis pigmentosa is the most prevalent form of congenital blindness. Using a retinitis pigmentosa mouse model, researchers have now shown that targeted activation of genes of similar function can compensate for the primary defect.

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  • Progress toward a treatment for Krabbe disease

    Aug 26, 2020 | 23:56 pm

    The inherited disease, which typically kills children before their second birthday, has no cure, but a new study in a canine model offers hope for an effective gene therapy with lasting results.

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  • Safer CRISPR gene editing with fewer off-target hits

    Jul 9, 2020 | 20:16 pm

    The CRISPR system is a powerful tool for the targeted editing of genomes, with significant therapeutic potential, but runs the risk of inappropriately editing ''off-target'' sites. However, a new study shows that mutating the enzyme at the heart of the CRISPR gene editing system can improve its fidelity.

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  • Scientists use nanoparticle-delivered gene therapy to inhibit blinding eye disease in rodents

    Jul 7, 2020 | 17:32 pm

    In experiments in rats and mice, two scientists -- an engineer and an ophthalmologist -- report the successful use of nanoparticles to deliver gene therapy for blinding eye disease. A uniquely engineered large molecule allows researchers to compact large bundles of therapeutic DNA to be delivered into the cells of the eye.

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  • Common inherited genetic variant identified as frequent cause of deafness in adults

    Jul 7, 2020 | 02:38 am

    A common inherited genetic variant is a frequent cause of deafness in adults, meaning that many thousands of people are potentially at risk, reveals new research.

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  • Duchenne muscular dystrophy: Substituting the next-best protein

    Apr 24, 2020 | 19:27 pm

    Children born with Duchenne muscular dystrophy have a mutation in the X-chromosome gene that would normally code for dystrophin, a protein that provides structural integrity to skeletal muscles. The loss of this protein causes severe symptoms, including deteriorating muscle strength beginning around the age of four. While there is no cure, a promising area of research has developed around the protein utrophin, which is approximately 80 percent identical to dystrophin and even takes its place early during muscle development.

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  • Diabetes reversed in mice with genetically edited stem cells derived from patients

    Apr 23, 2020 | 03:40 am

    Researchers have used induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes, transformed the stem cells into insulin-producing cells, used the CRISPR gene-editing tool to correct a defect that caused the diabetes, and implanted the cells into mice to reverse diabetes in the animals.

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  • Glaucoma could be successfully treated with gene therapy

    Apr 21, 2020 | 17:26 pm

    A new study has shown a common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients.

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  • Novel technology aims to improve treatment of neurological diseases

    Apr 18, 2020 | 03:29 am

    Researchers are developing new 'gene promoters' - which act like switches to turn genes on - for use with gene therapy, the delivery of new genes to replace ones that are faulty. The new promoters work especially well for brain and other neurological disorders and provide longer-lasting functionality compared to other promoters.

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  • Simulations show how to make gene therapy more effective

    Apr 17, 2020 | 17:45 pm

    Diseases with a genetic cause could be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human cells is difficult. A promising method for the delivery of such genes involves the use of DNA/lipid complexes (lipoplexes). Scientists have now used advanced simulations to investigate how these lipoplexes deliver DNA fragments into cells. The results can be used to improve their efficiency.

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  • Switching on a key cancer gene could provide first curative treatment for heart disease

    Apr 14, 2020 | 14:42 pm

    Researchers trying to turn off a gene that allows cancers to spread have made a surprising U-turn. By making the gene overactive and functional in the hearts of mice, they have triggered heart cell regeneration. Since adult hearts cannot usually repair themselves once damaged, harnessing the power of this gene represents major progress towards the first curative treatment for heart disease.

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  • New research suggests in-womb gene correction

    Apr 6, 2020 | 04:33 am

    New research led by hearing scientists suggests an avenue to treat and prevent intractable genetic disorders before birth. Researchers, working with mice, injected a specially designed synthetic molecule into the developing inner ear of fetal mice 12 days after fertilization. The study found that the technique corrected the expression of a mutated gene that causes Usher syndrome.

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  • Gene therapy reverses heart failure in mouse model of Barth syndrome

    Mar 9, 2020 | 21:52 pm

    Barth syndrome is a rare genetic disease in boys that can cause life-threatening heart failure and also weakens the skeletal muscles and the immune system. There is no specific treatment, but new research, involving new mouse models, shows the potential of a gene therapy approach in preventing and reversing cardiac dysfunction in Barth syndrome.

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  • Cells carrying Parkinson's mutation could lead to new model for studying disease

    Feb 27, 2020 | 20:42 pm

    Parkinson's disease researchers have used gene-editing tools to introduce the disorder's most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular chemistry that often goes awry in Parkinson's patients.

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  • New CRISPR base-editing technology slows ALS progression in mice

    Feb 26, 2020 | 19:05 pm

    With a new CRISPR gene-editing methodology, scientists have inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis -- a debilitating and fatal neurological disease for which there is no cure. The novel treatment slowed disease progression, improved muscle function and extended lifespan in mice with an aggressive form of ALS.

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  • Novel techniques for mining patented gene therapies offer promising treatment options

    Feb 7, 2020 | 20:17 pm

    Scientists are working to gain a better understand of the growing number of worldwide patented innovations available for gene therapy treatment.

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  • New gene therapy method improves vision in mice with congenital blindness

    Feb 7, 2020 | 15:54 pm

    Mice born blind have shown significant improvement in vision after undergoing a new gene therapy.

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