Glucan fingerprint as biomarker in Adult polyglucosan body disease (APBD)

Awardee: Berge Minassian

Institution: UT southwestern medical center

Grant Amount: $49,677.00

Funding Period: February 1, 2023 - January 31, 2024


Summary:

The central paradigm in APBD is excessive and abnormal glycogen in the central nervous system. While it is impossible to non invasively measure this glycogen, we can measure the breakdown products of it in the urine. We devised a technique to profile many of these glycogen breakdown products and showed evidence of being able to use such molecules in the urine of APBD model mice. The current grant will establish testing for other breakdown products in mouse models and patient samples.

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Exploring adjuvant immunotherapy to treat lymphangioleiomyomatosis

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Defining APBD pre-clinical biomarkers and assessing a therapy in an APBD mouse model.