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Novel gene therapy platform speeds search for ways to cure blindness
diagram of gene therapy for the eye showing injection into vitreous

Novel gene therapy platform speeds search for ways to cure blindness

A newly developed single-cell RNA sequencing technique enables researchers to quickly identify an optimal vector for delivering therapeutic genetic material to treat vision disorders, and perhaps other genetic conditions.

Katherine Unger Baillie

Four success stories in gene therapy

Four success stories in gene therapy

Jean Bennett and Albert Maguire of the Perelman School of Medicine developed a gene therapy to treat blindness in patients with retinal dystrophy caused by mutations in the RPE65 gene. “These people can now do things they never could have dreamed of doing, and they’re more independent and enjoying life,” said Bennett.

National Academy of Medicine welcomes two new members from Penn
Marylyn Ritchie and Sarah Tishkoff

Marylyn D. Ritchie and Sarah A. Tishkoff are Penn’s newest elected members of the National Academy of Medicine.

National Academy of Medicine welcomes two new members from Penn

The Perelman School of Medicine’s Marylyn D. Ritchie and PIK Professor Sarah A. Tishkoff are among 100 new members to be elected this year to the Academy, one of the highest honors in health and medicine.

Katherine Unger Baillie

A two-pronged approach to keep rheumatoid arthritis in check
Image showing body with shoulder, wrist, elbow, and hip joints in red indicating pain and inflammation

Painful inflammation characterizes rheumatoid arthritis, but a new study points to a possibly strategy to alleviate it: boosting levels of the protein DEL-1. 

A two-pronged approach to keep rheumatoid arthritis in check

A new study led by George Hajishengallis of the School of Dental Medicine shows that the protein DEL-1 could reduce the painful inflammation of RA in an animal model.

Katherine Unger Baillie

Imaging technology maps cells tied to inflammatory bowel disease
Microscopic multicolor image of a colon.

Multicolor image of a colon from a patient with ulcerative colitis stained by imaging mass cytometry. (Image: Courtesy of Ayano Kondo from the Kaestner Lab)

Imaging technology maps cells tied to inflammatory bowel disease

“Imaging mass cytometry” shows how cells tied to inflammatory bowel disease affect intestinal tissue, generating new theories for the progression of Crohn’s disease and ulcerative colitis.

From Penn Medicine News

Test predicts which patients with rare blood disease will respond to treatment
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)

Test predicts which patients with rare blood disease will respond to treatment

A Penn Medicine study identifies blood proteins that indicate which patients with idiopathic multicentric Castleman disease are most likely to benefit from the only FDA approved treatment for the disease, and uncovers an alternative.

From Penn Medicine News