DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy.
Since those first 2015 papers, dozens more have been published and multiple startups have launched, spurred by the hope that CRISPR could outperform the gene therapy approaches now nearing approval. One company was even bought out for over $200 million. Yet, with one exception, no gene-editing treatments for the rare muscle-wasting disease have entered the clinic and none appear particularly close.
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