Gene therapy shows promise for treating Niemann-Pick disease type C1

Wednesday, October 26, 2016

NIH mouse study could lead to human clinical trials.

For the first time, National Institutes of Health researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). The gene therapy involved inserting a functional copy of the NPC1 gene into mice with the disease; the treated animals were then found to have less severe NPC1 symptoms.

This detailed drawing of cells visualizes how the gene therapy involved inserting a functional copy of the NPC1 gene into mice with the disease.