A study in prenatal gene editing with DNA in utero
For the first time,scientists have performed prenatal gene editing to prevent a lethal metabolic disorder in laboratory animals, offering the potential to treat human congenital diseases before birth. Research published in Nature Medicinefrom the Perelman School of Medicine and the Children’s Hospital of Philadelphia offers proof-of-concept for prenatal use of a sophisticated, low-toxicity tool that efficiently edits DNA building blocks in disease-causing genes.
Using both CRISPR-Cas9 and base editor 3 (BE3) gene-editing tools, the team reduced cholesterol levels in healthy mice treated in uteroby targeting a gene that regulates those levels. They also used prenatal gene editing to improve liver function and prevent neonatal death in a subgroup of mice that had been engineered with a mutation causing a lethal liver disease.
“We used base editing to turn off the effects of a disease-causing genetic mutation,” says study co-leader Kiran Musunuru, an associate professor of cardiovascular medicine. “We also plan to use the same base-editing technique not just to disrupt a mutation’s effects, but to directly correct the mutation.”
Novel plant-based approach to a better, cheaper GLP-1 delivery system
Research led by Penn Dental’s Henry Daniell investigates the use of a lettuce-based, plant-encapsulated delivery platform as a new oral delivery of two GLP-1 drugs previously approved by the FDA in injectable form.
No brain, no gain: Neuronal activity enhances benefits of exercise
Research led by Penn neuroscientist J. Nicholas Betley and collaborators finds that hypothalamic neurons are essential for translating physical exertion into endurance, potentially opening the door to exercise-mimicking therapies.
In honor of Valentine's Day, and as a way of fostering community in her Shakespeare in Love course, Becky Friedman took her students to the University Club for lunch one class period. They talked about the movie "Shakespeare in Love," as part of a broader conversation on how Shakespeare's works are adapted.
In Becky Friedman’s English course Shakespeare in Love, undergraduate students analyze language, genre, and adaptation in the Bard’s plays through the lens of love.
Beating the heat: Designing cooling for bodies in motion
Dorit Aviv, director of Weitzman’s Thermal Architecture Lab, studies how humans, technology, and design intersect, paving the way for the development of novel approaches to cooling people efficiently.
