Article

Nanobodies as therapies for loss-of-function misfolding diseases

2025-04-17

Abstract excerpt

Misfolding diseases that result in loss of function represent a considerable burden for both individuals and society. Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder caused by mutations in the alanine:glyoxylate aminotransferase 1 (AGT) enzyme. The underlying molecular mechanisms causing PH1 are associated with protein misfolding (enhanced aggregation and mitochondrial mistargeting). The main therape...

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Literature Corpus work: a77e5b83-bd6b-561e-bf22-a7bdd1858446
DOI: 10.1101/2025.04.12.648492

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