Genetics

Vision researchers honored by End Blindness 2020

The Outstanding Achievement Prize highlights the contributions of the School of Veterinary Medicine’s Gustavo D. Aguirre and the Perelman School of Medicine’s Jean Bennett and Albert M. Maguire toward a gene therapy for a form of blindness.

Katherine Unger Baillie

Progress toward a treatment for Krabbe disease

The inherited disease, which typically kills children before their second birthday, has no cure, but a School of Veterinary Medicine study in a canine model offers hope for an effective gene therapy with lasting results.

Katherine Unger Baillie

Parasites and the microbiome

In a study of ethnically diverse people from Cameroon, the presence of a parasite infection was closely linked to the make-up of the gastrointestinal microbiome, according to a research team led by Penn scientists.

Katherine Unger Baillie



In the News


The Washington Post

FDA approves two sickle cell therapies, including first CRISPR medicine

Kiran Musunuru of the Perelman School of Medicine says that gene editing will be the biggest story of the century.

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Philadelphia Inquirer

More than 260,000 Penn Medicine patients have agreed to share their DNA for research, and the discoveries are just getting started

More than 260,000 people have signed up to participate in Penn Medicine BioBank, co-directed by Marilyn Ritchie and Dan Rader, which cross-references DNA with electronic health records to discover genetic variants of medical conditions.

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Philadelphia Inquirer

Jim Wilson has two new promising gene therapy studies, but says investment in the cutting-edge field has ‘gotten worse’

Jim Wilson of the Perelman School of Medicine has published two new studies supporting the promise of cutting-edge gene therapy, finding evidence that the genetic treatments can be beneficial for years without raising the risk of cancer.

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Smithsonian Magazine

Famed 5,300-year-old Alps Iceman was a balding middle-aged man with dark skin and eyes

Iain Mathieson of the Perelman School of Medicine says that the Iceman genome was one of the first ancient human genomes ever published.

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WHYY (Philadelphia)

Philly gene therapy companies tap into city’s workforce training program for lab techs

Penn Medicine is noted for spearheading gene therapy for cancer treatment, being the first in the nation to use a gene editing tool to combat cancer in 2019.

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Philadelphia Inquirer

These eight diseases are so rare that drug firms haven’t tried treating them with gene therapy. A $97 million program aims to help

Gene therapy for a rare form of blindness will be tested at Penn Medicine while gene therapy for a condition that causes skeletal deformities and seizures will be tested at the Children’s Hospital of Philadelphia.

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