EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
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Ornithine Transcarbamylase Deficiency, Hyperammonemia Due to
 - Cell Therapy Approaches

Ornithine transcarbamylase deficiency (OTCD) is a metabolic urea cycle disorder caused by impaired enzyme ornithine transcarbamylase function, resulting in ammonia accumulation in the blood, causing hyperammonemia.

Cell Therapy Approaches


Cell therapy utilizing hepatocytes or liver-derived stem/progenitor cell transplantation has the potential to relieve disease symptoms. Liver-derived stem/progenitor cells will differentiate into mature, healthy liver cells that can produce ornithine transcarbamylase enzyme and restore the normal urea cycle, thereby relieving disease symptoms.

Ornithine Transcarbamylase Deficiency, Hyperammonemia Due to