Doctors Used first Personalized Gene-editing Therapy on Baby
A team of doctors and scientists has successfully treated a rare genetic disorder using the first personalized gene-editing therapy. The results, published in The New England Journal of Medicine, were accompanied by an editorial from a former FDA gene-therapy regulator.
The patient in this landmark case was KJ, an infant diagnosed with CPS1 deficiency—a condition with a roughly 50% fatality rate in the first week of life.
CRISPR-Based Therapy Offers Hope for Devastating Genetic Disorder
Those who survive often face serious neurological damage, cognitive and developmental impairments, and may require liver transplants. KJ’s medical team created a personalized gene-editing therapy using CRISPR, a tool for altering human DNA.
Years of federally funded medical research enabled KJ’s successful gene repair, drawing on advances like CRISPR technology and human genome sequencing to identify the mutation.
This gene-editing method could help treat various genetic disorders. Although the FDA approved two CRISPR-based drugs for sickle cell disease, researchers see great potential for further advances. Furthermore, ongoing advancements promise to expand the applications of this technology even more. Therefore, continued research remains crucial to unlocking its full potential.
Read the original article on: Engadget
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