Potential impact of early pharmacological treatment on gene therapy outcomes in AADC deficiency

Affiliations

• ¹ Department of Women/Child Health and Urological Sciences, Sapienza-University of Rome, Italy; Unit of Child Neurology and Psychiatry, Department of Neuroscience/Mental Health, Azienda Ospedaliero Universitaria Policlinico, Umberto 1, Rome, Italy.
• ² Unit of Neurosurgery, Sapienza - University of Rome, Italy.
• ³ Department of Neuroscience, IRCSS Stella Maris, Pisa, Italy; Department of Clinical and Experimental Medicine-University of Pisa Department of Human Neuroscience, Sapienza University of Rome, Italy.
• ⁴ Unit of Child Neurology and Psychiatry, Department of Neuroscience/Mental Health, Azienda Ospedaliero Universitaria Policlinico, Umberto 1, Rome, Italy.
• ⁵ Department of Experimental Medicine, Sapienza- University of Rome, Italy.
• ⁶ Department of Neuroscience, IRCSS Stella Maris, Pisa, Italy.
• ⁷ Unit of Child Neurology and Psychiatry, Department of Neuroscience/Mental Health, Azienda Ospedaliero Universitaria Policlinico, Umberto 1, Rome, Italy. Electronic address: francesco.pisani@uniroma1.it.