The team of researchers at the Oregon Health and Science University (OHSU) in Portland – edited out a series of genes linked to an deadly inheritable disease. At the time, the disease in question wasn’t revealed, but now, thanks to the dramatic release of a peer-reviewed Nature study, the details have been made clear.
The disease in question is a type of heart condition – hypertrophic cardiomyopathy – that often kills except to the perfectly healthy people. It’s often symptomless until death suddenly takes the individual
The disease’s main effect is to thicken the muscular wall – the myocardium – to a point where it becomes stiff. Although with treatment, most people can live ordinary lives, this surprisingly common condition can lead to people getting anything from an irregular heartbeat to experiencing a sudden cardiac arrest.
Although this is at least the second attempt in the world to modify genetically viable human embryos using CRISPR, this study represents the first successful attempt to remove a disease in vivo.
Formerly, only in vitro fertilization (IVF) could minimize the risk that a child would inherit the potentially deadly gene. Although still at the proof-of-concept phase, this new experiment suggests that the near-future will feature a way not to just “pre-emptively cure” a person of the disease, but remove it from their offspring forever.
The team note that there’s room for improvement. Although their method ensured that 42 out of 58 embryos were free of the gene just five days post-incubation – a success rate of around 72 percent – they would ideally like to reach 100 percent effectiveness in the long run.
Importantly, they still must make sure that their gene-editing technique doesn’t cause inadvertent mutations elsewhere in the embryo. So far, though, so good – only one of these 42 embryos exhibited any signs of errant mutation.
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