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Getting gene therapy to the brain
Illustration of brain with DNA double helix

Crossing the blood-brain barrier to treat the whole brain has been a challenge for researchers aiming to treat inherited neurodegenerative disease. The results from a study in a large animal model offer “a big advance” in this pursuit, says John Wolfe of Penn Vet, Penn Medicine, and CHOP.

Getting gene therapy to the brain

Using a large animal model of genetic brain disease, researchers led by John H. Wolfe of the School of Veterinary Medicine, Perelman School of Medicine, and Children’s Hospital of Philadelphia delivered an effective treatment across the blood-brain barrier to correct the whole brain.

Katherine Unger Baillie

‘Self-eating’ stem cell process may be the key to new regenerative therapies
stem cells in autophagy

A translucently colored embryonic stem (ES) cell and its differentiating derivatives. (Image: Penn Medicine News)

‘Self-eating’ stem cell process may be the key to new regenerative therapies

A Penn study uncovers new roles of chaperone-mediated autophagy in how stem cells repair or regenerate damaged organs.

From Penn Medicine News

Interning virtually
Screenshot of a video call with 13 people

An open and engaging virtual orientation session set the tone for the Translational Research Internship Program, held online this year for the first time. (Image: Courtesy of Jessica German)

Interning virtually

The Translational Research Internship Program, offered by the Perelman School of Medicine’s Institute for Translational Medicine and Therapeutics Education Programs, provides mentorship for undergraduates as they complete a translational research project.

Katherine Unger Baillie

Rare genetic mutation leaves people at higher risk for multiple cancers
illustration of a microscopic gene and the isolated protein with the mutation.

Rare genetic mutation leaves people at higher risk for multiple cancers

Researchers find a new and previously undiscovered mutation in a particular gene associated with a higher risk of multiple kinds of cancer, and can lead to modified screenings for this particular gene protein.

John Infanti

Researchers find three distinct immune responses for sicker COVID-19 patients
Gloved hand in a lab dropping blood sample from a pipette into a petri dish.

nocred

Researchers find three distinct immune responses for sicker COVID-19 patients

Researchers from the Penn Institute of Immunology discovered three distinct immune responses to the SARS-CoV2 infection that could help predict the trajectory of disease in severe COVID-19 patients and may ultimately inform how to best treat them.

From Penn Medicine News