Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Open Access
- 12 June 2020
- journal article
- research article
- Published by Elsevier BV in Molecular Therapy - Methods & Clinical Development
- Vol. 17, 771-784
- https://doi.org/10.1016/j.omtm.2020.04.001
Abstract
No abstract availableFunding Information
- Association Française contre les Myopathies
- Conseil Régional des Pays de la Loire
This publication has 51 references indexed in Scilit:
- Single-Dose Gene-Replacement Therapy for Spinal Muscular AtrophyThe New England Journal of Medicine, 2017
- AAV.shRNA-mediated downregulation of ROCK2 attenuates degeneration of dopaminergic neurons in toxin-induced models of Parkinson's disease in vitro and in vivoNeurobiology of Disease, 2015
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia BThe New England Journal of Medicine, 2014
- AAV2-mediated striatum delivery of human CDNF prevents the deterioration of midbrain dopamine neurons in a 6-hydroxydopamine induced parkinsonian rat modelExperimental Neurology, 2013
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia BThe New England Journal of Medicine, 2011
- Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric DisordersMolecular Therapy, 2011
- Systemic Delivery of scAAV9 Expressing SMN Prolongs Survival in a Model of Spinal Muscular AtrophyScience Translational Medicine, 2010
- RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMNNature Biotechnology, 2010
- Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor NeuronsMolecular Therapy, 2009
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytesNature Biotechnology, 2008