The race is on to edit the DNA in our body to fight or prevent disease. Promising results from animal studies targeting the liver, muscles and the brain suggest that the CRISPR genome-editing method could revolutionise medicine, allowing us to treat or even cure a huge range of disorders.
The CRISPR genome-editing method was only developed in 2012, but it is proving so powerful and effective that around 20 trials in humans have already begun or will soon.
…Intellia Therapeutics of Cambridge, Massachusetts, for instance, is using fatty particles to deliver the CRISPR components to livers.
…[Jeffrey Chamberlain at the University of Washington] has been able to edit tissues all over the body using viruses. Earlier this year, his team successfully treated muscular dystrophy in mice by injecting them with an adeno-associated virus carrying DNA coding for the CRISPR components.
Nicole Deglon of Lausanne University Hospital in Switzerland and colleagues have developed a way to prevent the cutting protein lingering for too long when viruses are used for delivery…
Last month, her team showed that this system reduces off-target effects in a mouse study targeting the gene that causes Huntington’s disease.
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