Gene editing restores some sight in pair of children treated for blindness
CRISPR-Cas9 gene editing has been found to be safe and largely effective in addressing a form of inherited blindness in a group of patients that, for the first time, included children
CRISPR-Cas9 gene editing has been found to be safe and largely effective in addressing a form of inherited blindness in a group of patients that, for the first time, included children. In a multisite clinical trial called BRILLIANCE that included researchers from the Perelman School of Medicine and Children’s Hospital of Philadelphia (CHOP), 14 people—including two children under 17 years old—with leber congenital amaurosis (LCA), a form of blindness resulting from mutations in the CEO290 gene, received a single, surgical injection of a gene editing agent. Of those 14, nearly half reported measurable improvements in sight, including the two children according to the study, published in the New England Journal of Medicine (NEJM).
“Our patients are the first congenitally blind children to be treated with gene-editing, which significantly improved their ability to see things like daylight. Our hope is that the study will pave the road for treatments of younger children with similar conditions and further improvements in vision,” says Tomas S. Aleman, the Irene Heinz-Given and John LaPorte Research Professor in Ophthalmology at Penn Medicine with the Scheie Eye Institute and a pediatric ophthalmologist at CHOP who served as a site principal investigator and study co-author. “This trial represents a landmark in the treatment of genetic diseases, in specific, genetic blindness, by offering an important alternative treatment, when traditional forms of gene therapy, such as gene augmentation, are not an option.”
In Senegal, the ambitious Dakar Greenbelt project seeks to create an extensive network of ecological infrastructure in and around the city to sustainably address environmental concerns and enhance urban life. With support from David Gouverneur and Ellen Neises, Ph.D. candidate Rob Levinthal in the Weitzman School of Design led two courses that included a field trip to Dakar, that culminated in students presenting their visions for parts of the Greenbelt.
From a desert to an oasis: Penn engages in ambitious greening effort in the Sahel
Students from the Weitzman School of Design journeyed to Senegal to help with a massive ecological and infrastructural greening effort as part of their coursework. The Dakar Greenbelt aims to combat desertification and promote sustainable urban growth.
As part of an undergraduate course, Penn faculty and students curated an Arthur Ross Gallery exhibition of works from the Neumann family’s extensive collection of modern and contemporary art.
The University’s nexus for technology transfer supports researchers in their innovative efforts, from CAR T to mRNA advancements that have dramatically reshaped the world.
