Article

AAV-Mediated Dual-Gene Therapy Restores Metabolic Function in Mice with Propionic Acidemia

2026-03-07

Abstract excerpt

<h4>Background and Aims</h4> Propionic acidemia (PA) is a rare autosomal recessive disorder caused by mutations in PCCA or PCCB , which encode the two subunits of propionyl-CoA carboxylase (PCC). PCC deficiency causes toxic metabolite accumulation and multi-organ damage. Current management, including dietary restriction, pharmacological support, and liver transplantation, does not restore enzymatic activity. We...

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Literature Corpus work: 9b5cfe72-2402-52b4-a170-310b60628a4e
DOI: 10.64898/2026.03.06.709717

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