New NIH viral vector flips the script on sickle cell disease gene therapy
Researchers at the NIH have rolled out a new vehicle for sickle cell gene therapy with higher speeds and better horsepower, potentially allowing vastly more efficient gene transfer and a much larger carrying capacity. The best part? Unlike current sickle cell gene therapy models, the NIH one doesn’t have to drive in reverse.
In mice and monkeys, the new vehicle was up to 10 times more efficient and had a carrying capacity – the amount of DNA it can haul – of up to 6 times that of the conventional vectors currently deployed in gene therapy trials across the country. Most notably, the new vector can read the therapeutic gene sequence forward rather than reading them backward — a counter-intuitive trick researchers had used to overcome long-running barriers to gene therapy but which sacrificed efficiency. The results were published open access in Nature Communications.
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