The two-gene medication has been proven to recover motor functions in rats. After several months of treatment, rodents were able to use previously paralyzed limbs. Researchers at Kazan Federal University are now seeking pre-clinical trial investment.
People left blind by retinal degeneration have one option: electronic eye implants. UC Berkeley neuroscientists have developed an alternative: gene therapy that, in tests, restored vision in blind mice. A gene for green opsin delivered via virus gave blind mice enough sight to discern patterns on an iPad at a resolution sufficient for humans to read. Given existing AAV eye therapies already approved, this new therapy could be ready for clinical trials in three years.
It has been known for decades that cancer cells have an altered metabolism and it is seen in several biochemical pathways and in particular, in the way they get energy for their survival. Now, a new article describes an epigenetic injury found in human tumours which created this altered path to take energy from the cancer.
Ganglion cells in the eye generate noise as the light-sensitive photoreceptors die in diseases such as retinitis pigmentosa. Now, UC Berkeley neurobiologists have found a drug and gene therapy that can tamp down the noise, improving sight in mice with RP. These therapies could potentially extend the period of useful vision in those with degenerative eye diseases, including, perhaps, age-related macular degeneration.
Today, leading scientists and ethicists from seven countries have called for an international moratorium on the use of genetic editing to modify the human germline for clinical purposes.
A new biomarker that could be used to provide earlier diagnosis for a life limiting lung condition has been identified by researchers at the University of Bradford.
A second person has experienced sustained remission from HIV-1 after ceasing treatment, reports a paper led by researchers at UCL and Imperial College London. The case report, published in Nature and carried out with partners at the University of Cambridge and the University of Oxford, comes ten years after the first such case, known as the 'Berlin Patient.'
New research shows that long-lived parents produce better quality offspring. Researchers studied a gene associated with ageing in roundworms. They found that by reducing this gene's expression, they could not only more than double the worm's lifespan - but also improve the fitness of its offspring. The findings support an emerging new theory that we have genes that age us, and that shutting down these genes in later life could one day help us stay younger and healthier for longer.
Researchers at the National Cancer Institute use novel tools to reveal that cancer gene MYC causes global changes in gene activation, with subtle differences between individual cells.
The vast majority of patients with neurodegenerative disorders do not have specific gene mutations, but a single misbehaving protein -- called TDP-43 -- seems to be at the heart of these diseases. Pitt researchers have found a way to recreate and rescue TDP-43 pathology in a dish.