Table 3.

Approaches for evaluating cell therapy in WD

Assay	Usefulness
Serum ceruloplasmin	Not useful for evaluating success of cell therapy.
Serum or urine Cu levels	Of unknown or limited value for cell therapy.
Liver Cu content	Highly useful and specific. Difficult to sample by liver biopsy on multiple occasions. Potential for sampling errors owing to variable liver copper content related to areas with higher or lower numbers of transplanted cells
Molecular assays (e.g., ATP7B mRNA or ATP7B protein expression levels)	Highly useful and specific. Requires multiple liver samples. Potential sampling issues as above.
Liver histology and markers of tissue injury	Highly useful. Sampling issues as above.
Bile copper assays	Highly useful and specific. Bile may be collected by duodenal intubation.
Noninvasive reporter assays	Molecular imaging of ATP7B function has excellent potential. Reporter constructs could be introduced in transplanted cells to demonstrate extent of liver repopulation, (e.g., by imaging modalities).