Science Daily Gene Therapy News
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Safer CRISPR gene editing with fewer off-target hits
Jul 9, 2020 | 20:16 pmThe 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 pmIn 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 amA 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 pmChildren 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 amResearchers 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 pmA 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 amResearchers 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 pmDiseases 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 pmResearchers 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 amNew 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 pmBarth 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 pmParkinson'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 pmWith 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 pmScientists 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 pmMice 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 pmResearchers 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 pmDuchenne 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 pmResearchers 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 pmResearchers 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 pmResearchers 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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