KJ Muldoon, a baby boy with a rare genetic disorder, has thrived after receiving the first gene-editing CRISPR therapy tailored specifically for his unique mutation. At nearly ten months old, following three doses of the treatment, he is showing improvement, but his doctors state it is premature to declare a cure. Developed by an international team in just six months, this therapy represents a significant step forward in personalized medicine and gene therapy, potentially leading the way for other ultra-rare genetic diseases.
This truly is the future for all of these gene and cell therapies," says Arkasubhra Ghosh, who studies gene therapy at Narayana Nethralaya Eye Hospital in Bengaluru, India.
This is still really early days," says Rebecca Ahrens-Nicklas, a paediatrician at Children's Hospital of Philadelphia, about KJ's treatment.
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