Research graph
References from Advancing AADC deficiency therapy through MR-guided multisite delivery of AAV2-hAADC to dopaminergic and serotonergic pathways in the brainstem. Local targets link to admitted publications; unresolved targets remain external evidence.
Aromatic amino acid decarboxylase deficiency in twins
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Unresolved reference
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Aromatic L-amino acid decarboxylase deficiency in Taiwan
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Aromatic L-amino acid decarboxylase deficiency: clinical features, drug therapy and follow-up
10.1007/s10545-009-1076-1 · 2009 · External reference
Aromatic amino acid decarboxylase deficiency: Molecular and metabolic basis and therapeutic outlook
10.1016/j.ymgme.2019.03.009 · 2019 · External reference
AADC deficiency from infancy to adulthood: Symptoms and developmental outcome in an international cohort of 63 patients
10.1002/jimd.12247 · 2020 · External reference
Treatment of Parkinsonism With Levodopa
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Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency
10.1186/s13023-016-0522-z · 2017 · External reference
Aromatic L-Amino Acid Decarboxylase Gene Therapy Enhances Levodopa Response in Parkinson’s Disease
10.1002/mds.27993 · 2020 · External reference
Clinical and biochemical features of aromatic L-amino acid decarboxylase deficiency
10.1212/wnl.0b013e3181e620ae · 2010 · External reference
Biosynthesis of Catecholamines
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Storage and Release of Catecholamines
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Case report: Childhood epilepsy and borderline intellectual functioning hiding an AADC deficiency disorder associated with compound heterozygous DDC gene pathogenic variants
10.3389/fneur.2023.1284339 · 2023 · External reference
Gene therapy for aromatic L-amino acid decarboxylase deficiency
10.1126/scitranslmed.3003640 · 2012 · External reference
Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial. Lancet Child Adolesc
2017 · External reference
Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach
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Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC
10.1016/j.ymthe.2006.05.005 · 2006 · External reference
Efficient gene therapy-based method for the delivery of therapeutics to primate cortex
10.1073/pnas.0810682106 · 2009 · External reference
Safety and Tolerability of Magnetic Resonance Imaging-Guided Convection-Enhanced Delivery of AAV2-hAADC with a Novel Delivery Platform in Nonhuman Primate Striatum
10.1089/hum.2011.162 · 2012 · External reference
Glial-derived neurotrophic factor gene transfer for Parkinson’s disease: Anterograde distribution of AAV2 vectors in the primate brain
10.1016/j.nbd.2011.10.004 · 2012 · External reference
A biochemical and functional protein complex involving dopamine synthesis and transport into synaptic vesicles
10.1074/jbc.m109.054510 · 2010 · External reference
Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate
2014 · External reference
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
10.1038/s41467-021-24524-8 · 2021 · External reference
Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery
10.3171/2018.10.jns182169 · 2020 · External reference
Neurofilament-Light Chain as Biomarker of Neurodegenerative and Rare Diseases With High Translational Value
10.3389/fnins.2020.00579 · 2020 · External reference
Aromatic L-amino acid decarboxylase-immunoreactive structures in human midbrain, pons, and medulla
10.1016/j.jchemneu.2009.06.010 · 2009 · External reference
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
10.1212/wnl.0b013e3181c29356 · 2009 · External reference
Successful and safe perfusion of the primate brainstem: in vivo magnetic resonance imaging of macromolecular distribution during infusion
10.3171/jns.2002.97.4.0905 · 2002 · External reference
Prospective feasibility and safety assessment of surgical biopsy for patients with newly diagnosed diffuse intrinsic pontine glioma
10.1093/neuonc/noy070 · 2018 · External reference
Repeat convection-enhanced delivery for diffuse intrinsic pontine glioma
10.3171/2020.6.peds20280 · 2020 · External reference
Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial
10.1212/wnl.0000000000012952 · 2022 · External reference
Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson’s Disease
10.1002/mds.30193 · 2025 · External reference
A dynamic and multimodal framework to define microglial states
10.1038/s41593-025-01978-3 · 2025 · External reference
Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV
10.1016/j.ymthe.2003.12.005 · 2004 · External reference
Oxidative Stress and Dopaminergic Metabolism: A Major PD Pathogenic Mechanism and Basis of Potential Antioxidant Therapies
10.2174/1871527322666230609141519 · 2024 · External reference
Complexity of dopamine metabolism
10.1186/1478-811x-11-34 · 2013 · External reference
Hydroxyl radical formation via iron-mediated Fenton chemistry is inhibited by methylated catechols
10.1016/s0304-4165(98)00062-2 · 1998 · External reference
Evidence That Substantia Nigra Pars Compacta Dopaminergic Neurons Are Selectively Vulnerable to Oxidative Stress Because They Are Highly Metabolically Active
10.3389/fncel.2022.826193 · 2022 · External reference
Microglia Diversity in Healthy and Diseased Brain: Insights from Single-Cell Omics
10.3390/ijms22063027 · 2021 · External reference
Manufacturing and regulatory strategies for clinical AAV2-hRPE65
10.2174/156652310793180715 · 2010 · External reference
Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain
10.1038/mt.2008.111 · 2008 · External reference
Slow AAV2 clearance from the brain of nonhuman primates and anti-capsid immune response
10.1038/gt.2015.87 · 2016 · External reference
Safety Assessment of AAV2-hGDNF Administered Via Intracerebral Injection in Rats for Treatment of Parkinson’s Disease
10.1177/1091581820966315 · 2021 · External reference
Image-guided convection-enhanced delivery platform in the treatment of neurological diseases
10.1016/j.nurt.2007.10.064 · 2008 · External reference
Lead-OR: A multimodal platform for deep brain stimulation surgery
10.7554/elife.72929 · 2022 · External reference
Lead-DBS v3.0: Mapping deep brain stimulation effects to local anatomy and global networks
10.1016/j.neuroimage.2023.119862 · 2023 · External reference
The ANTsX ecosystem for quantitative biological and medical imaging
10.1038/s41598-021-87564-6 · 2021 · External reference
A reproducible evaluation of ANTs similarity metric performance in brain image registration
10.1016/j.neuroimage.2010.09.025 · 2011 · External reference
High-resolution mapping and digital atlas of subcortical regions in the macaque monkey based on matched MAP-MRI and histology
10.1016/j.neuroimage.2021.118759 · 2021 · External reference
