{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T17:28:25Z","timestamp":1790875705661,"version":"4.1.0"},"reference-count":26,"publisher":"Wiley","license":[{"start":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T00:00:00Z","timestamp":1782950400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T00:00:00Z","timestamp":1782950400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["movementdisorders.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Movement Disorders"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Intracerebral gene therapy is effective for amino acid decarboxylase (AADC) deficiency, but relationships between anatomical putaminal coverage, metabolic dynamics, and clinical recovery remain poorly understood.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>Assess safety, long\u2010term efficacy, and clinical\u2013radiological correlations in a genetically diverse European cohort of AADC deficiency.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Six patients received bilateral intraputaminal rAAV2\u2010hAADC infusion. Motor (GMFM\u201088), functional (CP\u2010CHILD, Vineland\u2010II), oculogyric crises (OGC), and dyskinesia outcomes were evaluated alongside magnetic resonance imaging (MRI) volumetric coverage and longitudinal 18F\u2010fluorodopa (18F\u2010DOPA) positron emission tomography (PET) parameters.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>All patients achieved genotype\u2010independent motor improvements and OGC reduction, with no serious adverse events. Clinical recovery did not correlate with the putaminal coverage volume, indicating a biological threshold effect. Transient dyskinesias coincided with an early 18F\u2010DOPA uptake peak (1\u20133\u2009months post\u2010operative), followed by clinical resolution and sustained activity, reflecting homeostatic synaptic plasticity.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>\n                      Eladocagene exuparvovec offers durable clinical benefits across diverse genotypes. Therapeutic efficacy and the subsequent neuroplasticity sequence seem to depend on reaching a critical threshold of dopamine production rather than on exhaustive anatomical coverage of the putamen. \u00a9 2026 The Author(s).\n                      <jats:italic>Movement Disorders<\/jats:italic>\n                      published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mds.70422","type":"journal-article","created":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T09:03:47Z","timestamp":1782983027000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Gene Therapy for\n                    <scp>Amino Acid Decarboxylase<\/scp>\n                    Deficiency: Clinical and Imaging Outcomes in a French Cohort"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0009-0005-0192-6205","authenticated-orcid":false,"given":"Cl\u00e9ment","family":"Dunoyer","sequence":"first","affiliation":[{"name":"Department of Neurosurgery Montpellier University Hospital  Montpellier France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5637-2004","authenticated-orcid":false,"given":"Ga\u00ebtan","family":"Poulen","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery Montpellier University Hospital  Montpellier France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marie\u2010C\u00e9line","family":"Fran\u00e7ois\u2010Heude","sequence":"additional","affiliation":[{"name":"Department of Neuropediatrics Montpellier University Hospital, University of Montpellier  Montpellier 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