Doctors claimed Thursday that a US newborn with a rare ailment has become the first patient in history to receive treatment using a customised gene-editing technology, which gives hope to others with unknown conditions. KJ Muldoon, a 9-and-a-half-month-old boy with large blue eyes and plump cheeks, is the little pioneer.
He was diagnosed with CPS1 deficiency, a rare and dangerous illness, shortly after birth. It is brought on by a mutation in a gene that codes for an enzyme essential to liver function, which stops those who have it from getting rid of some hazardous waste products from their metabolism.
Given the dire outlook, medical professionals proposed a novel approach: a customised therapy that would repair the baby’s DNA using a method akin to a pair of molecular scissors. This method is known as Crispr-Cas9, and its developers were awarded the 2020 Nobel Prize in Chemistry.
According to the boy’s father, he and his spouse had to make a hard choice. Our kid is ill. We must either give him this medication that has never been administered to anybody before or obtain a liver transplant, right?” Kyle Muldoon stated.
Ultimately, they decided to treat the boy with an infusion explicitly designed for him to rectify his genetic mutation, which is a mistake in one of the billion DNA letters that make up the human genome.
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