Nature Communications (Jul 2021)
Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons
- Toni S. Pearson,
- Nalin Gupta,
- Waldy San Sebastian,
- Jill Imamura-Ching,
- Amy Viehoever,
- Ana Grijalvo-Perez,
- Alex J. Fay,
- Neha Seth,
- Shannon M. Lundy,
- Youngho Seo,
- Miguel Pampaloni,
- Keith Hyland,
- Erin Smith,
- Gardenia de Oliveira Barbosa,
- Jill C. Heathcock,
- Amy Minnema,
- Russell Lonser,
- J. Bradley Elder,
- Jeffrey Leonard,
- Paul Larson,
- Krystof S. Bankiewicz
Affiliations
- Toni S. Pearson
- Department of Neurological Surgery, University of California San Francisco
- Nalin Gupta
- Department of Neurological Surgery, University of California San Francisco
- Waldy San Sebastian
- Department of Neurological Surgery, University of California San Francisco
- Jill Imamura-Ching
- Department of Neurological Surgery, University of California San Francisco
- Amy Viehoever
- Department of Neurology, University of California San Francisco
- Ana Grijalvo-Perez
- Department of Neurology, University of California San Francisco
- Alex J. Fay
- Department of Neurology, University of California San Francisco
- Neha Seth
- Department of Rehabilitative Services, University of California San Francisco
- Shannon M. Lundy
- Department of Pediatrics, University of California San Francisco
- Youngho Seo
- Department of Radiology and Biomedical Imaging, University of California San Francisco
- Miguel Pampaloni
- Department of Radiology and Biomedical Imaging, University of California San Francisco
- Keith Hyland
- Medical Neurogenetics Laboratories
- Erin Smith
- Therapy Services, St. Louis Children’s Hospital
- Gardenia de Oliveira Barbosa
- School of Health and Rehabilitation Sciences, The Ohio State University
- Jill C. Heathcock
- School of Health and Rehabilitation Sciences, The Ohio State University
- Amy Minnema
- Department of Neurological Surgery, The Ohio State University
- Russell Lonser
- Department of Neurological Surgery, The Ohio State University
- J. Bradley Elder
- Department of Neurological Surgery, The Ohio State University
- Jeffrey Leonard
- Department of Neurological Surgery, The Ohio State University
- Paul Larson
- Department of Neurological Surgery, University of California San Francisco
- Krystof S. Bankiewicz
- Department of Neurological Surgery, University of California San Francisco
- DOI
- https://doi.org/10.1038/s41467-021-24524-8
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 12
Abstract
Aromatic L-amino acid decarboxylase deficiency (AADC) is a rare neurodevelopmental disorder. Here the authors describe a clinical trial of MR-guided delivery of AAV2-AADC for the treatment of AADC.
