This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...

Earlier this year, researchers at CHOP and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a rare liver disorder. KJ Muldoon ...

For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not make ...

Researchers report early but encouraging results on an experimental gene-editing treatment for high cholesterol.

Gene therapy is defined as the treatment of disease by transfer of genetic material into cells, to prevent, treat and potentially even cure a disease. Gene therapies can work by several mechanisms: 1) ...

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function ...

A new experimental gene therapy has successfully restored immune system function in children born with ADA-SCID, a rare and potentially fatal genetic immune disorder. Developed by researchers at UCLA, ...

Moorfields Eye Hospital in London made international headlines last week. Doctors announced they had saved the sight of four young children suffering from a rare genetic condition that rapidly causes ...