Title : Gene Therapy in a Mouse Model of Niemann-Pick Disease Type C1.

Pub. Date : 2021 Jun

PMID : 33256498






1 Functional Relationships(s)
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Protein Name
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1 Accumulated unesterified cholesterol in liver was reduced in the AAV-treated <i>Npc1<sup>-/-</sup></i> mice. Cholesterol NPC intracellular cholesterol transporter 1 Homo sapiens