gene therapy Although a variety of diseases can be effectively treated, size poses a major problem for some debilitating diseases, such as muscular dystrophy. Genes that...
It is the only approved ocular gene therapy and targets two forms of specific genetic mutations. hereditary retinal diseaseA real-world study in the UK has shown...
Spinal muscular atrophy (SMA) is a severe neurological disease for which there is currently no cure, although current treatments can alleviate symptoms. In the search for...
Because the majority of genetic studies have focused on people of European descent, our understanding of how genes affect the health of other populations is limited....
Drug development is typically slow. It can take decades to go from the basic research discovery that underpins a new drug through clinical trials to the...
When Michael Pirovolakis received personalized gene therapy in a single-patient clinical trial at SickKids Hospital in March 2022, the course of his disease changed dramatically. Michael...
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