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A High-Content Screening Assay for Small Molecules That Stabilize Mutant Triose Phosphate Isomerase (TPI) as Treatments for TPI Deficiency

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Published in 2021 at "SLAS Discovery"

DOI: 10.1177/24725552211018198

Abstract: Triose phosphate isomerase deficiency (TPI Df) is an untreatable, childhood-onset glycolytic enzymopathy. Patients typically present with frequent infections, anemia, and muscle weakness that quickly progresses with severe neuromusclar dysfunction requiring aided mobility and often respiratory… read more here.

Keywords: triose phosphate; phosphate isomerase; tpi; tpi treatments ... See more keywords