Systemic AAV9 Gene Therapy Improves The Lifespan of Mice with Niemann-Pick Disease, Type C1 (IMAGE)

NIH/National Human Genome Research Institute

Systemic AAV9 Gene Therapy Improves The Lifespan of Mice with Niemann-Pick Disease, Type C1

Caption

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.

Credit

Darryl Leja, NHGRI

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