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• Breakthrough in gene therapy coming out of Penn Medicine and the Children’s Hospital of Philadelphia Thursday, May 15, 2025

  Landmark research using CRISPR-based gene editing technology, developed at Penn, can precisely correct disease-causing variants in the human genome to treat patients with rare diseases for whom no medical treatments are available. CRISPR technology was used to treat KJ, an infant born with a rare metabolic disease known as severe carbamoyl phosphate synthetase 1 (CPS1) deficiency. KJ’s medical team, including Rebecca Ahrens-Nicklas (right) and Kiran Musunuru (left) of the Perelman School of Medicine, targeted KJ’s specific variant of CPS1. KJ, who spent the first several months of his life in the hospital, received the first dose of his customized therapy in February. The lifesaving treatment was administered safely, and he is now growing well and thriving. “We want each and every patient to have the potential to experience the same results we saw in this first patient, and we hope that other academic investigators will replicate this method for many rare diseases and give many patients a fair shot at living a healthy life,” Musunuru says. “The promise of gene therapy that we’ve heard about for decades is coming to fruition, and it’s going to utterly transform the way we approach medicine.”

  Read the full story in Penn Today. 

• UPenn MSGC Program Celebrates 5 Years at Penn - and a New Certificate Program for Advanced Research Training for GCs Thursday, May 1, 2025

  The MSGC program is celebrating the graduation of it's 5th class at UPenn, following 23 at Arcadia University!), and the growth and development that has occurred since the program moved to it's new home in 2019.  An article in Penn Medicine News explores the future of genetic counseling, with a spotlight on Penn's Advanced Research Training for Genetic Counselors (ART-GC) certificate program, which is funded by a generous grant from the Warren Alpert Foundation.  Click the title to read the full story.

• Katalin Karikó and Drew Weissman, Penn’s historic mRNA vaccine research team, win 2023 Nobel Prize in Medicine Monday, October 2, 2023

  The University of Pennsylvania messenger RNA pioneers whose years of scientific partnership unlocked an understanding of how to modify mRNA to make it an effective therapeutic—enabling a platform used to rapidly develop lifesaving vaccines amid the global COVID-19 pandemic—have been named winners of the 2023 Nobel Prize in Physiology or Medicine. They become the 28th and 29th Nobel laureates affiliated with Penn, and join nine previous Nobel laureates with ties to the University of Pennsylvania who have won the Nobel Prize in Medicine.

  Nearly three years after the rollout of mRNA vaccines across the world, Katalin Karikó, an adjunct professor of neurosurgery in Penn’s Perelman School of Medicine, and Drew Weissman, the Roberts Family Professor of Vaccine Research in the Perelman School of Medicine, are recipients of the prize announced this morning by the Nobel Assembly in Solna, Sweden.