Gene therapy

The Children's Tumor Foundation's mission is to drive research, expand knowledge, and advance care for the NF community.

New gene therapy breakthrough from the University of Hawai'i offers safer, efficient treatments for genetic disorders like Hemophilia.

The pediatric patients, with a rare neurodegenerative disease, were treated with bluebird bio’s Skysona to slow the progression of neurologic dysfunction. Six patients developed myelodysplastic syndrome and one patient developed acute myeloid leukemia.

A novel non-viral gene therapy for discogenic back pain (DBP).

David Liu's team at the Broad Institute has achieved a major milestone in gene editing, creating a system that could treat a wide range of genetic disorders by inserting healthy genes directly into their native locations

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Over the past few years the discipline has evolved in significant ways.

Gene therapy in a nutshell