A genetic disease needs a genetic answer.

Step 1

A letter is missing

NPC1 is a gene. In NPC, both copies are mutated, so the cell cannot make enough working NPC1 protein to move cholesterol out of the lysosome.

Step 2

We write the letter again

Scientists build a correct messenger RNA — a temporary instruction that says how to make NPC1 protein. It does not permanently edit DNA.

Step 3

A tiny ship delivers it

The mRNA is wrapped in a lipid nanoparticle, a fat bubble that can travel through the body and enter cells.

Step 4

The cell builds the missing protein

Once inside, the cell’s own machinery reads the mRNA and assembles NPC1 protein. Cholesterol can start leaving the lysosome.

Step 5

Doses may need to be repeated

mRNA is temporary. The hope is that regular doses would keep enough NPC1 protein in place — a functional treatment at the root of the disease.

The Iowa partnership

Life 4 Liam partners with Dr. Mark Schultz and the Schultz Laboratory at the University of Iowa Stead Family Children’s Hospital. The lab is researching lipid-nanoparticle mRNA that can restore NPC1 protein.

Visit the Schultz Laboratory

This is research, not an approved medicine. It is the path this foundation was built to fund.

The roadmap

mRNA first. Prime editing next.

The Iowa work is not a dead end. The same science carries forward. We go from a functional cure that can keep our children alive, to a complete cure that rewrites the gene.