Indian-origin doctor leads gene editing breakthrough in Philadelphia

Baby KJ, born with a rare genetic disorder, received a custom gene editing therapy and is now growing and thriving, thanks to Dr Kiran Musunuru.

Dr. Kiran Musunuru / Penn Medicine

In a groundbreaking medical development, Dr. Kiran Musunuru, an Indian-origin physician and professor at Penn Medicine, has played a central role in successfully treating an infant with a rare genetic condition using a customized CRISPR-based gene editing therapy.

The treatment, developed in collaboration with researchers at the Children’s Hospital of Philadelphia (CHOP), marks the first known case of a bespoke gene editing therapy tailored to a single patient with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency.

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