Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

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Medicine, Neurology, Neuroscience +17

Consensus Paper: The Cerebellum's Role in Movement and Cognition

Leonard F. Koziol, Deborah Ely Budding, Nancy C. Andreasen et al. · The Cerebellum · 2013 · 1.1K citations · Full text

Life Sciences, Neurobiology, Neuroscience +12

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Florian Eichler, Christine Duncan, Patricia L. Musolino et al. · New England Journal of Medicine · 2017 · 595 citations

Medicine, Hematology, Neurology +16

Qualitative alteration in substrate specificity of mitochondrial monoamine oxidase in brain

Journal of Neurochemistry · 1976 · 445 citations

Life Sciences, Biochemistry, Endocrinology +9

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial

Stéphane Palfi, Jean Marc Gurruchaga, G. Ralph et al. · The Lancet · 2014 · 436 citations

Medicine, Neuroscience, Pharmacology +16