UPenn GTV PhD candidate and genome editor 🧬

Philadelphia, PA
Joined May 2025
.@SecKennedy - thank you so much for your broad support for gene editing and gene therapies as well as recognizing the importance of Baby KJ’s story and the potential that CRISPR base editing therapies hold for patients with severe genetic diseases. I’m thrilled you have publicly recognized the Herculean efforts that made KJs therapy possible, from the physicians @CHOP like @kiranmusunuru and Rebecca Ahrens-Nicklas to the technological innovation in @davidrliu and @BKleinstiver labs, and especially the coordination and cooperation of the regulators @US_FDA that made this possible. The gene editing medicine KJ received, a CRISPR adenine base editor was invented and developed in @davidrliu lab @Harvard with government grants from the NIH. This life-saving medicine, and so many future technologies, could not and will not be possible without government grants such as the one that supported this work, but are now shut off by this administration. So PLEASE ask this administration to stop attacking our academic institutions and restore government funding to @Harvard (where again, KJ’s medicine was created!!) We cannot have stories like KJ’s without full and unwavering support from the US government. Please help, please.
Thursday morning, I had the privilege of speaking with the Muldoon family, whose 10-month-old son, baby KJ, was discharged Wednesday from @ChildrensPhila. Baby KJ was diagnosed with a rare urea cycle disorder and received a first-of-its-kind personalized CRISPR gene-editing therapy—customized specifically for his unique mutation. Thanks to the extraordinary expertise and compassion of the team at CHOP and the researchers who developed this pioneering treatment, baby KJ now has a second chance at life. The Muldoon family’s courage is nothing short of inspiring, and I offer my deepest gratitude to everyone at CHOP and beyond who made this medical milestone possible. This isn’t just a medical success story—it’s a powerful glimpse into the future of personalized medicine. I remain firmly committed to working with @NIHDirector_Jay, @DrMakaryFDA, @DrOzCMS, and others to ensure that: • HHS continues to invest in cutting-edge research that drives breakthrough therapies • Our regulatory frameworks keep pace with rapid innovation • No family is ever denied access to life-saving treatments because of cost Breakthroughs like this are why we do the work.
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A few weeks ago we introduced you to KJ, who made medical history as the first patient to receive a personalized gene editing therapy. After almost a year at CHOP, KJ is now at home with his family. 🎓 Tune in to NBC's @TODAYshow tomorrow morning to hear from his family!
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Sarah Grandinette retweeted
Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. nyti.ms/4j3xGs8
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CHOP & @PennMedicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments. Learn more about KJ & the future of personalized medicine: ms.spr.ly/6015SZLAx.
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So happy to have helped make this personalized gene editing therapy a reality for KJ! Hopefully, this workflow can be applied to more N-of-1 patient variants to treat severe genetic diseases.
In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by @US_FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe, rapidly progressing genetic disease... all in an astounding 7 months. Best of all, the infant patient shows apparent benefit. Congratulations to @kiranmusunuru, Rebecca Ahrens-Nicklas, and other team members for this heroic and inspiring effort, which has implications for the hundreds of millions of patients that suffer from thousands of genetic diseases. drive.google.com/file/d/1Jfk…
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