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Molecular Therapy. Advances|May 15, 2026
Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstemLluis Samaranch, Clemens Neudorfer, Matthew T Rocco, et al.Nature Communications|July 13, 2021
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neuronsToni S Pearson, Nalin Gupta, Waldy San Sebastian, et al.Biorxiv : the Preprint Server for Biology|December 17, 2025
Efficient <i>in vivo</i> mammalian neuron editing using peptide-mediated CRISPR enzyme deliveryBrigette D Manohar, Meika Travis, Vikas Munjal, et al.Nature|April 8, 2021
The NIH Somatic Cell Genome Editing programKrishanu Saha, Erik J Sontheimer, P J Brooks, et al.Pageof 12