AADC Activity in Brain Evident 2 Years After Gene Therapy, Trial Data Show

AADC Activity in Brain Evident 2 Years After Gene Therapy, Trial Data Show

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AADC Activity in Brain Evident 2 Years After Gene Therapy, Trial Data Show

A viral vector-delivered gene therapy can restore the activity of the AADC enzyme in people with AADC deficiency for at least two years, research drawing on clinical trial data shows. Findings were in the study “Dopaminergic restoration of prefrontal cortico-putaminal network in gene therapy for aromatic L-amino acid decarboxylase deficiency,” published in Brain Communications. AADC is caused by mutations in the gene DDC. These mutations render the body unable to make the AADC (aromatic L-amino acid decarboxylase) enzyme,…

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