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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

Could psychedelics simultaneously treat chronic pain and depression?
Ahmad Hammo

nocred

Could psychedelics simultaneously treat chronic pain and depression?

This summer, Ahmad Hammo, a rising third-year student in the School of Engineering and Applied Science, is conducting a pilot study to explore psilocybin’s potential as a therapy for chronic pain and the depression that often accompanies it. 

Liana F. Wait