Life 4 Liam
mRNA packaged and protein restored in cells
The Schultz laboratory packaged NPC1 mRNA into a particle that can enter cells and restore the missing NPC1 protein. That opened the door to dosing studies and animal work.
Research
From a packaged particle to a living model.
The community funded the first year of Iowa work. The lab packaged NPC1 mRNA, restored protein in cells, and began animal studies. The road is still long. It is no longer theoretical.
$63,000 to Schultz Lab for the mouse work
By December 2023 the community had raised $63,000 for Dr. Mark Schultz’s laboratory at the University of Iowa Stead Family Children’s Hospital. That gift funded the mouse studies of the mRNA gene-therapy path — a viable treatment packaged, tested in cells, and moved into animal work.
The work continues — every day still counts
While two FDA-approved medicines arrived for NPC, Life 4 Liam is still chasing a genetic solution: regular mRNA doses that could let children make the protein they were born without. Updates will appear here as the Iowa work moves forward.
Cover paper in Molecular Therapy Nucleic Acids
Results from that mouse work will be published as the cover paper of the September 2026 issue of Molecular Therapy Nucleic Acids. The science we funded is leaving the lab notebook and entering the literature.