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QY202305)

10.1136/gut.2005.065557 343

The salvage pathway, which is predominant in mammals, recycles NAM derived from food intake and as a byproduct of NAD + consuming enzymes such as silent information regulator 2 proteins (Sirtuins), poly (ADP-ribose) polymerase 1 (PARP1), CD38 and Sterile Alpha and TIR Motif Containing 1 (SARM1)

Because these cells have been genetically modified, they can express the missing or faulty gene by secreting the gene product which is a protein or enzyme into the brain where it can be taken up by defective neurons, thus correcting the underlying deficiency causing the disease