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Molecular Therapy, ISSN 1525-0016, 02/2019, Volume 27, Issue 2, pp. 465 - 478
Adeno-associated virus-mediated gene replacement is emerging as a safe and effective means of correcting single-gene mutations affecting the CNS. AAV-mediated... 
AAV | neurodegeneration | progranulin | frontotemporal dementia | neuronal ceroid lipofuscinosis | brain gene therapy | CNS gene therapy | MEDICINE, RESEARCH & EXPERIMENTAL | FRONTOTEMPORAL DEMENTIA | PHENOTYPES | FEATURES | LIPOFUSCINOSIS | NEURITE OUTGROWTH | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | GENETICS & HEREDITY | GENE-THERAPY | MICE | MUTATIONS | VECTORS | EXPRESSION
Journal Article
Blood, ISSN 0006-4971, 10/2015, Volume 126, Issue 15, pp. 1777 - 1784
Journal Article
Nucleic Acids Research, ISSN 0305-1048, 12/2014, Volume 42, Issue 21, pp. 13440 - 13451
Journal Article
01/2019, ISBN 1085614794
Huntington’s disease (HD) is a fatal, genetic neurodegenerative disease that shares many features with other common neurological disorders, including early... 
Neurosciences | Molecular biology | Biomedical engineering
Dissertation
Human Molecular Genetics, ISSN 0964-6906, 12/2018, Volume 27, Issue 24, pp. 4303 - 4314
Abstract Polyglutamine (polyQ) repeat diseases are a class of neurodegenerative disorders caused by CAG-repeat expansion. There are diverse cellular mechanisms... 
BIOCHEMISTRY & MOLECULAR BIOLOGY | GENETICS & HEREDITY | 3-NITROPROPIONIC ACID | PARASPECKLES | BINDING-SITES | PROTEINS | EXPRESSION | DOMAINS | NUCLEAR-BODY | MICRORNA | DEGENERATION | P53 | Index Medicus | General
Journal Article
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