Aavrh.10-mnpc2-ha

1. Aavrh.10-mnpc2-ha decreases liver cholesterol in mice.

   “significant reduction of liver cholesterol and sphingomyelin levels in treated Npc2-/- mice.”

   Animalsin Npc2-/- miceAAVrh.10-mNpc2-HAliver

   Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2

   Experimental neurology·12 Apr 2018

More on Liver cholesterol

1. Aavrh.10-mnpc2-ha decreases purkinje cell death in mice.

   “reduced Purkinje cell death, decreased gliosis”

   Animalsin Npc2-/- miceAAVrh.10-mNpc2-HAbrain

   Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2

   Experimental neurology·12 Apr 2018

1. Aavrh.10-mnpc2-ha improves performance in behavioral tasks in mice.

   “improved performance in behavioral tasks.”

   Animalsin Npc2-/- miceAAVrh.10-mNpc2-HA

   Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2

   Experimental neurology·12 Apr 2018

1. Aavrh.10-mnpc2-ha extends lifespan in mice.

   “administration of AAVrh.10-mNpc2-HA significantly extended life-span.”

   Animalsin Npc2-/- miceAAVrh.10-mNpc2-HA

   Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2

   Experimental neurology·12 Apr 2018