EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
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All Diseases > Disease Card

Ornithine Transcarbamylase Deficiency
 - 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