Science Daily Gene Therapy News
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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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Gene therapy shown to offer long-term benefits for people with Haemophilia A
Jan 6, 2020 | 18:35 pmA 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 pmScientists 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 pmResearchers 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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High-tech method for uniquely targeted gene therapy developed
Dec 13, 2019 | 15:25 pmNeuroscientists have developed new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.
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Unique case of disease resistance reveals possible Alzheimer's treatment
Nov 4, 2019 | 17:28 pmDefying the odds, an individual at high risk for early-onset Alzheimer's disease remained dementia-free for many years beyond what was anticipated. A new study led researchers to suggest that a gene variant may be the key, perhaps providing a new direction toward developing a treatment.
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New gene therapy for epilepsy provides on-demand release of endogenous substance
Oct 30, 2019 | 16:00 pmScientists have developed a new therapeutic concept for the treatment of temporal lobe epilepsy. It represents a gene therapy capable of suppressing seizures at their site of origin on demand. Having been shown to be effective in an animal model, the new method will now be optimized for clinical use.
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Discovery in monkeys could lead to treatment for blindness-causing syndrome
Oct 25, 2019 | 20:53 pmA genetic mutation that leads to a rare, but devastating blindness-causing condition called Bardet-Biedl Syndrome has been discovered in monkeys for the first time. The finding offers a promising way to develop gene and cell therapies that could treat people with the condition, which leads to vision loss, kidney disfunction, extra fingers or toes, and other symptoms.
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Deleting a liver enzyme lowers the health risk of sweet treats (at least in mice)
Oct 24, 2019 | 16:58 pmHepatic insulin resistance, caused by diets high in sugar and fat, can lead to type 2 diabetes. Researchers found that the Elovl6 gene plays a key role in hepatic insulin resistance. Deleting Elov6 in liver cells causes a rise in a specific ceramide lipid that protects mice from hepatic insulin resistance due to excessive dietary sugar. The findings could help efforts to find a targeted treatment for the condition.
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When added to gene therapy, plant-based compound may enable faster, more effective treatments
Oct 17, 2019 | 22:22 pmToday's standard process for administering gene therapy is expensive and time-consuming -- a result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem. Scientists believe they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.
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In-office gene therapy for wet age-related macular degeneration is coming
Oct 11, 2019 | 13:47 pmGene therapy is showing promise for one of the most common causes of blindness.
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