Life 4 Liam
mRNA gene therapy
The Life 4 Liam approach: teach the cell to make the missing NPC1 protein using messenger RNA.
How it works
Children with NPC1 cannot make enough working NPC1 protein. mRNA therapy packages a correct instruction into a tiny fat bubble called a lipid nanoparticle. The cell reads that instruction and builds NPC1 protein so cholesterol can leave the lysosome.
Life 4 Liam partners with Dr. Mark Schultz at the University of Iowa Stead Family Children’s Hospital to develop this path. If it works, regular doses could functionally treat the root problem.
Who it is for
Not yet a standard clinical treatment. This is the research Life 4 Liam is funding.
What it does not do
It is not an approved medicine today. It has not yet completed the full path of animal studies, human trials, and FDA review.
What this treatment does
Four short pictures of the idea. Play through, or tap a step. This is a teaching picture, not a medical simulation.
Step 1 of 4 · The problem
The instruction for NPC1 is broken
Both copies of the gene are mutated, so the cell cannot make enough working NPC1 protein.
Official information
Educational only. Ask an NPC specialist what is right for a specific child or adult. Official sites and FDA pages are the source of prescribing information.