What are the limits of gene therapy? “There are no currently available FDA-approved gene therapy treatments for overactive bladder,” reads this press release. And that’s true. But is it, like, a problem? Urovant Sciences believes so, which is why it licensed just such a gene therapy for overactive bladder. It’s called hMaxi-K, and it targets...
Tag: <span>Gene Therapy</span>
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Gene therapy restores sense of smell in mice
Re-expressing a protein critical for the detection and perception of odors restores the function of the olfactory system in a genetic mouse model of lost hair-like cellular structures known as cilia, according to research published in JNeurosci. This may represent a promising therapeutic strategy for a group of human diseases that can cause loss of smell. Credit: Green...
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Researchers cure type 2 diabetes and obesity in mice using gene therapy
July 10, 2018, Autonomous University of Barcelona A research team from the UAB led by Professor Fatima Bosch has managed to cure obesity and type 2 diabetes in mice using gene therapy. The research team at CBATEG (UAB). Credit: UAB A single administration of an adeno-associated viral vector (AAV) carrying the FGF21 (Fibroblast Growth Factor 21) gene, resulted in genetic...
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Solution to 50-year-old mystery could lead to gene therapy for common blood disorders
UNSW Sydney scientist Professor Merlin Crossley. Credit: UNSW In a landmark study that could lead to new therapies for sickle cell anaemia and other blood disorders, UNSW Sydney-led researchers have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of foetal haemoglobin. The research solves a 50-year-old mystery...
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CRISPR enhances gene therapy to fight inherited diseases
Shown is a transmission electron microscope image of adenovirus particles. A new study, in mice, combined molecular tools — adenovirus and CRISPR — to improve gene therapy. Credit: CDC/G. William Gary Jr Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a “bad” gene...
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Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable
UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective. The research was published in the journal ACS Nano by senior author Paul...
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Gene therapy shows promise against blood-clotting disease
This Saturday, Dec. 2, 2017 photo provided by Jay Konduros, left, shows him and his brother, Bill, at Jay’s home in Cambridge, Ontario, Canada. The brothers, who have hemophilia, were involved in a gene therapy study for their condition. Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year...
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New gene therapy transplantation technique could improve treatment of neurodegenerative diseases
A reconstruction of a sagittal brain section of a mouse that received transplantation of HSPCs (green) through intra-cerebral ventricle injection. The image shows an abundance of progeny cells from the transplanted HSPCs. A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach to treating...
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More than a quarter of patients with a common type of deadly brain tumor were alive 3 years after undergoing new gene therapy, promising clinical trial reveals
Results were found after treating patients with recurrent high-grade glioma, the most common type of adult brain tumor It’s the type US Senator John McCain was diagnosed with earlier this year The cancer infiltrates into the normal brain tissue and is often incurable Median survival was 14.4 months, compared with 8 months typically seen New...
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Gene therapy protects against age-related cognitive and memory deficits
From left to right: researchers of the UAB Institute of Neurosciences Miguel Chillón, Assumpció Bosch, Lydia Giménez-Llort and Àngela Sánchez. Researchers from the Institute of Neurosciences at the Universitat Autònoma de Barcelona (INc-UAB) and the Vall d”Hebron Research Institute (VHIR) are the first to demonstrate that regulation of the brain’s Klotho gene using gene...