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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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