Epiliepsy currents/Epilepsy currents · 2019 · 16 citations · 51 references
MedicineGeneticsNeuroscienceEpilepsyGene DeliveryGene TherapyEpilepsy GeneticsGenetic EpilepsiesImmunogenicity Of Gene TherapiesIn Vivo Gene TherapyNeurological Gene TherapySafety Of Gene TherapyEpilepsy Gene TherapyGene-targeted Therapy
Gene therapy has recently advanced to the level of standard of care for several diseases. However, its application to neurological disorders is still in the experimental phase. In this review, we discuss recent advancements in the field that provide optimism on the possibility to have first-in-human studies for gene therapy of some forms of epilepsy in the not so distant future.
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Kevin D. Foust, Emily Nurre, Chrystal L. Montgomery et al. · Nature Biotechnology · 2008 · 1.4K citations · Full text