It is essential that gene therapy research for genetic disorders receives funding and donations, because if there is success in one of these diseases, there can possibly be a cure in other diseases.

In laboratories around the world, scientists are finally cracking illnesses that once looked untouchable, from inherited ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

David Fajgenbaum leads a research lab at the University of Pennsylvania dedicated to studying Castleman disease, a rare illness that nearly killed him five times. The physician-researcher runs a ...

Olivia Burtwistle is a 12-year-old girl in search of a cure for Batten disease. — -- Olivia Burtwistle grew as a typically curious and energetic child until soon after her fourth birthday, when ...

For decades, immunologists have explored CAR-T cell therapy as an effective tool to fight blood cancer. Increasingly, CAR-T cells are being explored as a potential silver bullet for treating ...

For the three out of every 100,000 children who are born with Batten disease, the diagnosis is one of the most devastating that a family can receive. Emily Blackburn, 32, found out in March 2023 that ...

For this week’s Open Questions column, Dhruv Khullar is filling in for Joshua Rothman. When David Fajgenbaum was a twenty-five-year-old medical student, at the University of Pennsylvania, he started ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...