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University of Iowa
Stead Family
Children's Hospital
Children with Niemann Pick Type-C are missing the NPC1 proteins which are responsible for breaking down and clearing out of cholesterol from the cells of the body. If the cholesterol builds up enough, the cell will inevitably die. When this happens in the brain and nervous system it causes neurodegeneration.
The new gene therapy that we are researching & developing leverages new advancements in mRNA technology and medicines to help the body produce the missing NPC1 proteins so the cells can break down and clear out the cholesterol.
If this gene therapy is successful children with Niemann Pick Type-C would be functionally cured of the disease by receiving regular doses of the treatment.
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