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Genetics

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

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.

‘In vivo’ RNA-based gene editing model for blood disorders developed
Microscopic rendering of mRNA gene editing.

Image: iStock/Ozgu Arslan

‘In vivo’ RNA-based gene editing model for blood disorders developed

Researchers from Penn Medicine and The Children’s Hospital of Philadelphia show that gene editing tools can be delivered via lipid nanoparticles, which would reduce cost and increase access to cutting-edge gene therapies.

Alex Gardner

Katalin Susztak hunts for a cure for kidney disease
Katalin Susztak

Katalin Susztak, professor of internal medicine, nephrology, and genetics at the Perelman School of Medicine.

(Image: Penn Medicine News)

Katalin Susztak hunts for a cure for kidney disease

Throughout her career, the professor of internal medicine, nephrology, and genetics has had a profound impact on the way kidney disease is identified, prevented, and managed.

From Penn Medicine News

Three things to know about BRCA mutations in men
A man sits with a doctor holding an iPad.

Image: Courtesy of Penn Medicine News

Three things to know about BRCA mutations in men

Kara Maxwell, director of the Men & BRCA Program at the Basser Center, is bridging the knowledge gap about how BRCA mutations affect men.

From Penn Medicine News