2024
DOI: 10.1007/s40272-024-00627-9
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Advances in Immune Tolerance Induction in Enzyme Replacement Therapy

Aslı İnci,
Fatih Süheyl Ezgü,
Leyla Tümer

Abstract: Inborn errors of metabolism (IEMs) are a group of genetic diseases that occur due to the either deficiency of an enzyme involved in a metabolic/biochemical pathway or other disturbances in the metabolic pathway including transport protein or activator protein deficiencies, cofactor deficiencies, organelle biogenesis, maturation or trafficking problems. These disorders are collectively significant due to their substantial impact on both the well-being and survival of affected individuals. In the quest for effec… Show more

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