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Portnoff JB, Henley MW
This is an inherited genetic disease called pyroglutamic aciduria which manifests as hemolytic anemia and metabolic acidosis, causing neurological disorders and vulnerability to bacterial infections, and which can be classified as mild, moderate or severe depending on the severity of the symptoms
Proteolysistargeting chimeras (PROTACs) are capable of selectively degrading target proteins within cells
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