ArticleEMBO molecular medicine2021
Gene therapy in the putamen for curing AADC deficiency and Parkinson's disease.
Article in EMBO molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.
- Mapping the research of mitochondria and Parkinson's disease: a bibliometric analysis.Frontiers in neurology · 2024Pooled it
- Intraputaminal AAV9 co-delivery of AADC and GDNF in severe AADC deficiency: first-in-human pediatric dose-escalation study.Science China. Life sciences · 2026Article
- Segmentation and structural connectivity of the putamen for targeted convection enhanced drug delivery in Parkinson's disease: a tractography-based approach.Frontiers in neuroscience · 2026Article
- Parkinson's Disease: From Bench to Bedside-Advancements in Diagnosis and Therapeutics using Pharmacogenomic Approach.Current gene therapy · 2026Review
- Identification of Hub Genes and Prediction of Interacting Chemicals in Parkinson's Disease Using Bioinformatics.Current medicinal chemistry · 2026Article
- The CRISPR-Cas9 knockout DDC SH-SY5Y in vitro model for AADC deficiency provides insight into the pathogenicity of R347Q and L353P variants: a cross-sectional structural and functional analysis.The FEBS journal · 2025Article
- In vivo applications and toxicities of AAV-based gene therapies in rare diseases.Orphanet journal of rare diseases · 2025Review
- Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases.Genes & diseases · 2025Review
- Current clinical applications of AAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- BAG3's dual roles in Parkinson's disease and cardiomyopathy: benefit or liability?Acta neuropathologica · 2024Article
- Gene Therapy for Parkinson's Disease Using Midbrain Developmental Genes to Regulate Dopaminergic Neuronal Maintenance.International journal of molecular sciences · 2024Review
- From lab bench to hope: a review of gene therapies in clinical trials for Parkinson's disease and challenges.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Review
- Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations.Molecular therapy. Methods & clinical development · 2024Article
- Patient selection considerations for AADC deficiency gene therapy.Annals of the Child Neurology Society · 2024Review
- Gene therapy for neurotransmitter-related disorders.Journal of inherited metabolic disease · 2024Review
- Potential Therapeutic Approach using Aromatic l-amino Acid Decarboxylase and Glial-derived Neurotrophic Factor Therapy Targeting Putamen in Parkinson's Disease.Current gene therapy · 2024Review
- PET reporter systems for the brain.Trends in neurosciences · 2023Review
- Intraputaminal Gene Delivery in Two Patients with Aromatic L-Amino Acid Decarboxylase Deficiency.Movement disorders clinical practice · 2023Article
- Compound Heterozygosis in AADC Deficiency and Its Complex Phenotype in Terms of AADC Protein Population.International journal of molecular sciences · 2022Review
- Breakthrough News in Adenoviral Vector-Mediated AADC Gene Therapy: Lessons from the Success in AADC Deficiency and Possible Future Applications.Movement disorders clinical practice · 2022Article
Corrections and comments
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Authors and funding
10 authors at 7 institutions in 5 countries.
Funding
Abstract
This commentary provides an overview of the putamen as an established target site for gene therapy in treating aromatic l-amino acid decarboxylase (AADC) deficiency and Parkinson's disease, two debilitating neurological disorders that involve motor dysfunction caused by dopamine deficiencies. The neuroanatomy and the function of the putamen in motor control provide good rationales for targeting this brain structure. Additionally, the efficacy and safety of intraputaminal gene therapy demonstrate that restoration of dopamine synthesis in the putamen by using low doses of adeno-associated viral vector serotype 2 to deliver the hAADC gene is well tolerated. This restoration leads to sustained improvements in motor and nonmotor symptoms of AADC deficiency and improved uptake and conversion of exogenous l-DOPA into dopamine in Parkinson's patients.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.