Doctors used CRISPR to rewrite a baby's DNA and fix a rare liver disorder

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Doctors used CRISPR to rewrite a baby's DNA and fix a rare liver disorder - Business Standard

Quick Summary:

Kiran Musunuru, MD, PhD, MPH, ML, MRA, (left) and Rebecca Ahrens-Nicklas, MD, PhD, (right) led the group of researchers from CHOP and Penn who developed a personalised treatment for baby KJ.

The treatment involved “base editing”—a highly specific method that alters a single DNA letter—and was delivered directly to KJ’s liver using lipid nanoparticles.

KJ was born with a condition called carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare and severe disorder that prevents the body from safely processing ammonia.