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Baby Is Healed With World's First Personalized Gene-Editing Treatment
Scientists have successfully treated a 9.5-month-old boy with an ultra-rare genetic disorder using the world's first personalized gene-editing therapy. The patient, identified as KJ, has CPS1 deficiency -- a condition affecting just one in 1.3 million babies that prevents proper ammonia processing and is often fatal.
The breakthrough treatment, detailed in the New England Journal of Medicine, uses base editing technology to correct KJ's specific DNA mutation. The therapy delivers CRISPR components wrapped in fatty lipid molecules that protect them in the bloodstream until they reach liver cells, where they make the precise edit needed.
After three infusions, KJ now eats normal amounts of protein and has maintained stable ammonia levels even through viral illnesses that would typically cause dangerous spikes. His weight has increased from the 7th to 40th percentile. Dr. Peter Marks, former FDA official, called the approach "one of the most potentially transformational technologies" because it could be rapidly adapted for thousands of other rare genetic diseases without lengthy development cycles.
of this story at Slashdot.
#baby #healed #with #world039s #first
Baby Is Healed With World's First Personalized Gene-Editing Treatment
Scientists have successfully treated a 9.5-month-old boy with an ultra-rare genetic disorder using the world's first personalized gene-editing therapy. The patient, identified as KJ, has CPS1 deficiency -- a condition affecting just one in 1.3 million babies that prevents proper ammonia processing and is often fatal.
The breakthrough treatment, detailed in the New England Journal of Medicine, uses base editing technology to correct KJ's specific DNA mutation. The therapy delivers CRISPR components wrapped in fatty lipid molecules that protect them in the bloodstream until they reach liver cells, where they make the precise edit needed.
After three infusions, KJ now eats normal amounts of protein and has maintained stable ammonia levels even through viral illnesses that would typically cause dangerous spikes. His weight has increased from the 7th to 40th percentile. Dr. Peter Marks, former FDA official, called the approach "one of the most potentially transformational technologies" because it could be rapidly adapted for thousands of other rare genetic diseases without lengthy development cycles.
of this story at Slashdot.
#baby #healed #with #world039s #first
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