Intraputaminal Gene Delivery in Two Patients with Aromatic L‐Amino Acid Decarboxylase Deficiency

BACKGROUND: Aromatic l‐amino acid decarboxylase deficiency (AADCD) is a rare, early‐onset, dyskinetic encephalopathy mostly reflecting a defective synthesis of brain dopamine and serotonin. Intracerebral gene delivery (GD) provided a significant improvement among AADCD patients (mean age, ≤6 years)....

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Detalles Bibliográficos
Autores principales: François‐Heude, Marie‐Céline, Poulen, Gaetan, Flamand Roze, Emmanuel, Nguyen Morel, Marie‐Ange, Gras, Domitille, Roch‐Toreilles, Isabelle, Quintard, Adeline, Baroux, Gaelle, Meyer, Pierre, Coubes, Philippe, Milesi, Christophe, Cambonie, Gilles, Baleine, Julien, Sola, Chrystelle, Delye, Bénédicte, Dimopoulou, Evgenia, Sanchez, Stéphanie, Gasnier, Mathieu, Touati, Souad, Zamora, Alberto, Pontal, Daniel, Leboucq, Nicolas, Kouyoumdjian, Virginie, Lebasnier, Adrien, Sanquer, Sylvia, Mariano‐Goulart, Denis, Roujeau, Thomas, Roubertie, Agathe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2023
Materias:
Brief Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10187009/
https://www.ncbi.nlm.nih.gov/pubmed/37205256
http://dx.doi.org/10.1002/mdc3.13685

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10187009/
https://www.ncbi.nlm.nih.gov/pubmed/37205256
http://dx.doi.org/10.1002/mdc3.13685

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