'A cause for celebration': New gene editing tool offers promise of treating many genetic diseases

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A study published Wednesday in the journal Nature, demonstrates a tool that can correct the kind of single-letter genetic mistakes that cause thousands of diseases.

This baseball fan stands 3 feet tall and weighs just 19 pounds. Josiah Viera is a young Cardinals fan diagnosed with the rare aging disease Progeria. But that doesn't stop him from motivating his favorite baseball players during spring training.A woman's gloved arm reaches down into a plastic tub and carefully gets hold of a mouse. Caught on video, the woman speaks in a coaxing voice as the tiny animal shivers.

In this case, Liu and his colleagues repaired a gene that, when mutated, causes progeria, an ultra-rare disease leading to premature aging. The average lifespan of someone with progeria is just 14, and they die of conditions such as heart disease that usually kill people decades older. Instead, a base editor tool works like a pencil, capable of erasing and rewriting a single letter mistake.

 

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