Doudna Says CRISPR Treated Baby KJ in 8 Months as Delivery Hurdles Still Limit Wider Use
Updated
Updated · Quanta Magazine · Jun 11
Doudna Says CRISPR Treated Baby KJ in 8 Months as Delivery Hurdles Still Limit Wider Use
3 articles · Updated · Quanta Magazine · Jun 11
Summary
Baby KJ, born in August 2024 with a rare metabolic disorder, received a customized CRISPR treatment within eight months and now appears to be thriving, Doudna said.
Three doses targeted his liver using existing CRISPR tools and a delivery method adapted from COVID-vaccine technology, showing a one-patient therapy could be built without new basic research.
Doudna said the biggest barrier to broader use is delivery beyond the liver—to tissues such as the lung, muscle and brain—along with the cost of scaling rare-disease treatments.
She also pointed to wider medical potential, including one-time cholesterol-editing therapies; Verve, pursuing that approach, was bought by Eli Lilly last year.
On ethics, Doudna called the 2018 embryo-editing case in China shocking and medically unnecessary, arguing CRISPR’s expansion in medicine, agriculture and climate applications still requires strong public oversight.