Here the idea is mechanical: deliver a working copy of the gene that codes for the missing enzyme into the patient’s cells, so those cells can make the enzyme themselves.

A one-time treatment path can put that working gene in through a carefully controlled clinic infusion. Cells then produce the missing enzyme tool so the harmful sugar can be broken down.

The product is not “a vitamin for the brain.” The product is an instruction set: gene in → enzyme made → less harmful buildup. Change any link (wrong gene, no delivery to the right tissues, immune wipeout) and the benefit fails.

“One-time” matters because cells that keep the working instructions can keep making enzyme without a daily lifelong enzyme bottle — while specialized monitoring still follows.