Personalized CRISPR treatment used on baby with genetic disease |
Doctors used personal gene therapy to treat an infant with a deadly genetic disease in a medical first.
KJ Muldoon was born in August 2024, and DNA sequencing revealed he had inherited two gene mutations that prevented him from breaking down proteins in food. That leads to a buildup of ammonia that destroys the liver and can cause neurological damage with lifelong consequences.
His condition, known as CPS1 deficiency, is one of a group of metabolic diseases with no cure. Half of all babies born with the condition die in their first week of life.
Patients who survive are usually treated with drugs to reduce the ammonia in their bodies, but........