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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.


  • 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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  • Six patients with rare blood disease are doing well after gene therapy clinical trial

    Jan 29, 2020 | 15:14 pm

    Researchers have reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD - which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan -- had to rely on bone marrow donations for a chance at remission.

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  • New gene correction therapy for Duchenne muscular dystrophy

    Jan 27, 2020 | 19:48 pm

    Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers have developed a gene therapy that may provide permanent relief for those suffering from DMD.

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  • Mechanism for how common gene therapy vectors enter cells

    Jan 23, 2020 | 17:59 pm

    Researchers have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types -- the most commonly used viral vectors for in vivo gene therapy. The researchers identified that GPR108, a G protein-coupled receptor, served as a molecular 'lock' to the cell. The discovery could one day enable scientists to better direct AAV gene transfers to specific tissues.

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  • Drug profiling and gene scissors open new avenues in immunotherapy

    Jan 21, 2020 | 18:40 pm

    Researchers have discovered ways to boost CAR T-cell therapy.

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  • Nanoparticles deliver 'suicide gene' therapy to pediatric brain tumors growing in mice

    Jan 8, 2020 | 15:03 pm

    Researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a ''suicide gene'' to pediatric brain tumor cells implanted in the brains of mice. The poly(beta-amino ester) nanoparticles, known as PBAEs, were part of a treatment that also used a drug to kill the cells and prolong the test animals' survival.

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  • Gene therapy shown to offer long-term benefits for people with Haemophilia A

    Jan 6, 2020 | 18:35 pm

    A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK.

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  • New technology allows control of gene therapy doses

    Dec 24, 2019 | 16:13 pm

    Scientists have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing. The feat offers gene therapy designers what may be the first viable technique for adjusting the activity levels of their therapeutic genes.

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  • For CRISPR, tweaking DNA fragments before inserting yields highest efficiency rates yet

    Dec 23, 2019 | 18:29 pm

    Researchers have now achieved the highest reported rates of inserting genes into human cells with the CRISPR-Cas9 gene-editing system, a necessary step for harnessing CRISPR for clinical gene-therapy applications. By chemically tweaking the ends of the DNA to be inserted, the new technique is up to five times more efficient than current approaches.

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