"Late last year, dozens of researchers spanning thousands of miles banded together in a race to save one baby boy's life. The result was a world first: a cutting-edge, gene-editing therapy fashioned for a single person, and produced in a record-breaking six months. Now, baby KJ Muldoon's doctors are gearing up to do it all over again, at least five times over."
"The groundbreaking clinical trial, described on 31 October in the American Journal of Human Genetics, will deploy an offshoot of the CRISPRCas9 gene-editing technique called base editing, which allows scientists to make precise, single-letter changes to DNA sequences. The study is expected to begin next year, after its organizers spent months negotiating with US regulators over ways to simplify the convoluted path a gene-editing therapy normally has to take before it can enter trials."
Dozens of researchers rapidly developed a personalized gene-editing therapy for baby KJ using base editing, producing the treatment within six months. A clinical trial will apply base-editing to at least five additional patients and aims to shorten development times further. Organizers negotiated with US regulators to simplify the usual complex path to trials. Base editing enables precise single-letter DNA changes, offering targeted correction of mutations causing rare diseases. Clinical activities are expanding, including efforts by a Center for Pediatric CRISPR Cures at UC Berkeley and UCSF. Families of children with rare diseases seek clearer timelines for access to individualized therapies.
Read at www.nature.com
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