British drugmaker GlaxoSmithKline made headlines last year when it won approval for its gene therapy Strimvelis in Europe. But, due to a small patient population and high price tag, the drug has only been used once. So far, despite higher levels of safety and efficacy than previous iterations, the new wave of gene therapies still...
Tag: <span>Gene Therapy</span>
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Dogs with Duchenne Treated with Gene Therapy
Like humans, some golden retrievers develop Duchenne muscular dystrophy (DMD), a hereditary muscle wasting condition that begins early in life. Using gene therapy, scientists were able to restore muscle function in dogs with the disease, according to a study published today (July 25) in Nature Communications. Researchers injected microdystrophin, a shortened version of the dystrophin gene that individuals with...
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New gene therapy treatment routes for motor neurone disease uncovered in new study
Scientists investigating the genetic causes and altered functioning of nerve cells in motor neuron disease (MND) have discovered a new mechanism that could lead to fresh treatment approaches for one of the most common forms of the disease. The team, based in the Sheffield Institute for Translational Neuroscience (SITraN), investigated a mutation in one particular...
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Gene therapy could ‘turn off’ severe allergies
A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland. A team led by Associate Professor Ray Steptoe at the UQ Diamantina Institute has been able to ‘turn-off’ the immune response which causes allergic reaction in animals. “When someone has an...
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Injured bones reconstructed by gene and stem cell therapies
This illustration shows the bone-tissue engineering technique developed by Cedars-Sinai investigators. ‘Endogenous MSCs’ refers to stem cells from a patient’s bone. The ‘BMP gene’ is a gene that promotes bone repair. A Cedars-Sinai-led team of investigators has successfully repaired severe limb fractures in laboratory animals with an innovative technique that cues bone to regrow...
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New gene therapy for vision loss proven safe in humans
A fundus photo showing intermediate age-related macular degeneration. In a small and preliminary clinical trial, Johns Hopkins researchers and their collaborators have shown that an experimental gene therapy that uses viruses to introduce a therapeutic gene into the eye is safe and that it may be effective in preserving the vision of people with...
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Type 1 diabetes cured in mice using gene therapy
Researchers from the University of Texas Health Science Center in San Antonio have found a way to cure type 1 diabetes in mice. It is hoped that the novel technique – which boosts insulin secretion in the pancreas – will reach human clinical trials in the next 3 years. Researchers have successfully used gene therapy...
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A New Gene Therapy Cure Just Treated Its First-Ever Patient
Rarefied Cures This March, child with severe combined immune deficiency (SCID) became the second commercial gene therapy patient ever. The patient started receiving a drug called Strimvelis from GlaxoSmithKline — a drug that appears to be able to cure the rare, inherited immune disorder SCID. The drug was approved for use in commercial gene therapy in May of 2016,...
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Testing the Efficacy of New Gene Therapies More Efficiently
Using a new cellular model, innovative gene therapy approaches for the hereditary immunodeficiency Chronic Granulomatous Disease can be tested more rapidly and more cost-effectively in the lab for their efficacy. A team of researchers from the University of Zurich and the Children’s Hospital Zurich successfully achieved this using the ‘gene-scissor’ CRISPR/Cas9 technology. The aim is...
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Immunotherapy, gene therapy combination shows promise against glioblastoma
“Devastating” and “dismal.” That’s how leading researchers describe the present outlook for malignant brain tumors. The median survival rate for patients with glioblastoma multiforme, or GBM, is a mere 14.2 months. New research out of the University of Michigan supports combining two approaches to fight back against gliomas: attacking the tumor with gene therapy while...