AAV9-NPC1 gene therapy

1. AAV9-NPC1 gene therapy decreases motor decline in mice.

   “The Npc1-/- mice that received a single dose of AAV9-CamKII-NPC1 as neonates (2.6 × 1011GC) or at weaning (1.3 × 1012GC), and the mice that received a single dose of AAV9-EF1a-NPC1 at weaning (1.2 × 1012GC), exhibited an increased life span, characterized by delayed weight loss and diminished motor decline.”

   Animalsin Npc1-/- mice

   Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

   Human molecular genetics·1 Jan 2017

1. AAV9-NPC1 gene therapy decreases cholesterol in mice.

   “Cholesterol storage and Purkinje neuron loss were also reduced in the central nervous system of AAV9 treated Npc1-/- mice.”

   Animalsin Npc1-/- mice

   Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

   Human molecular genetics·1 Jan 2017

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