A single injection reverses blindness in patient with rare genetic disorder
A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye. The clinical trial was conducted by researchers at the Scheie Eye Institute in the Perelman School of Medicine. Results of the case, detailed in a paper published in Nature Medicine, show that the treatment led to marked changes at the fovea, the most important locus of human central vision.
The treatment was designed for patients diagnosed with Leber congenital amaurosis (LCA)—an eye disorder that primarily affects the retina—who have a CEP290 mutation, which is one of the more commonly implicated genes in patients with the disease. Patients with this form of LCA suffer from severe visual impairment, typically beginning in infancy.
“Our results set a new standard of what biological improvements are possible with antisense oligonucleotide therapy in LCA caused by CEP290 mutations,” says co-lead author Artur V. Cideciyan,a research professor of ophthalmology. “Importantly, we established a comparator for currently-ongoing gene editing therapies for the same disease, which will allow comparison of the relative merits of two different interventions.”
OpenAI, DeepSeek, and Google vary widely in identifying hate speech
Neil Fasching and Yphtach Lelkes of the Annenberg School for Communication have found dramatic differences in how large language models classify hate speech, with especially large variations for language about certain demographic groups, raising concerns about bias and disproportionate harm.
Understanding order to disorder at the atomic scale opens possibilities for next-generation electronic devices
A Penn team has developed insight into the chemical and geometric mechanisms underlying the synthesis of new 2D materials, paving the way for next-gen devices, biomedical applications, and cleaner, quicker energy conversion and storage.
(From left) Kevin B. Mahoney, chief executive officer of the University of Pennsylvania Health System; Penn President J. Larry Jameson; Jonathan A. Epstein, dean of the Perelman School of Medicine (PSOM); and E. Michael Ostap, senior vice dean and chief scientific officer at PSOM, at the ribbon cutting at 3600 Civic Center Boulevard.
Reimagined Penn Medicine facility set to usher in a bold new era of Immune Health discovery
3600 Civic Center Boulevard will bring together key researchers and technologies in Immune Health, the Colton Center for Autoimmunity, and infectious diseases to drive breakthrough science.
