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World’s first patient treated with personalized CRISPR gene editing therapy through CHOP and Penn Med collaboration
Kiran Musunuru and Rebecca Ahrens-Nicklas holding KJ in the hospital.

Penn Medicine’s Kiran Musunuru and Rebecca Ahrens-Nicklas holding KJ post infusion.

(Image: Courtesy of Children’s Hospital of Philadelphia)

World’s first patient treated with personalized CRISPR gene editing therapy through CHOP and Penn Med collaboration

A landmark study from CHOP and Penn Medicine showcases the power of customized gene editing therapy to treat a patient with a rare metabolic disease

Matt Toal

5 min. read

First new subtype of Castleman disease discovered in 45 years
David Fajgenbaum in his lab.

David Fajgenbaum is an assistant professor of medicine at the Perelman School of Medicine at the University of Pennsylvania and associate director of patient impact in the Penn Orphan Disease Center. He also leads the Castleman Disease Research Program.

(Image: Courtesy of Penn Medicine)

First new subtype of Castleman disease discovered in 45 years

A new study co-authored by Penn Medicine’s David Fajgenbaum expands the spectrum of the rare disorder, which will help diagnose and treat patients caught between existing classification systems.

2 min. read

Penn-made AI tool matched a patient’s rare disease with a common inflammation drug. He’s been in remission for over two years

Penn-made AI tool matched a patient’s rare disease with a common inflammation drug. He’s been in remission for over two years

Every Cure, a nonprofit founded by David Fajgenbaum of the Perelman School of Medicine, has received $108 million in federal contracts and private financing to identify promising disease-medication matches and make recommendations for further clinical study.

Researchers create genetic map tied to kidney disease
microscopic rendering of DNA strands.

Image: iStock/TanyaJoy

Researchers create genetic map tied to kidney disease

The creation of the most complete map of more than 1,000 genes that influence kidney function could help experts diagnose and design targeted treatments for kidney disease.

Alex Gardner

Breakthroughs in gene editing and expression control with mvGPT
Tyler Daniel using a pipette in a bioengineering lab.

Sherry Gao, Tyler Daniel (pictured), and their coauthors have developed a new tool that can simultaneously and independently edit multiple genes and regulate their expression.

(Image: Bella Ciervo)

Breakthroughs in gene editing and expression control with mvGPT

Penn Engineers have created a gene editing tool that can address different genetic diseases in the same cell.

Ian Scheffler