Review
Open access
Aug 2026
Current Status of Cellular and Gene-Based Therapies for Congenital Metabolic Disorders: A Review
Congenital metabolic disorders often lead to irreversible organ damage beginning during fetal life or shortly after birth. Advances in prenatal diagnostics have enabled earlier identification of these conditions, creating opportunities for prenatal therapeutic intervention. Preclinical studies have demonstrated that pr...
Angelika Masiarz, Pola Kosteczko, Magdalena Mazur et al.
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