AIMC Topic: Ornithine Carbamoyltransferase

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Biochemical Characterization of Disease-Associated Variants of Human Ornithine Transcarbamylase.

ACS chemical biology
Human ornithine transcarbamylase deficiency (OTCD) is the most common ureagenesis disorder in the world. OTCD is an X-linked genetic deficiency in which patients experience hyperammonemia to varying degrees depending on the severity of the genetic mu...

Therapeutic enzyme engineering using a generative neural network.

Scientific reports
Enhancing the potency of mRNA therapeutics is an important objective for treating rare diseases, since it may enable lower and less-frequent dosing. Enzyme engineering can increase potency of mRNA therapeutics by improving the expression, half-life, ...