Table 5 Fluorogenic substrate for MPS enzymes

From: Ready-to-use iPSC-derived microglia progenitors for the treatment of CNS disease in mouse models of neuropathic mucopolysaccharidoses

Disease

Gene/Enzyme

Involved degradation pathway

Artificial Fluorogenic Substrate

MPS I (Scheie, Hurler syndrome)

IDUA (Ī±-L-Iduronidase)

DS, HS

4-Methylumbelliferyl-a-L-iduronide

MPS VII (Sly syndrome)

GUSB (Ī²-Glucuronidase)

DS, HS,

4-Methylumbelliferyl-beta-D-glucuronide

MPS IIIA (Sanfilippo A syndrome)

SGSH (Sulfamidase)

HS

-

MPS IIIB (Sanfilippo B syndrome)

NAGLU (N-acetylglucosaminidase)

HS

4-Methylumbelliferyl-N-acetyl-alpha-D-glucosaminide

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