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Online ISSN : 1884-7668
Print ISSN : 0029-0831
ISSN-L : 0029-0831
<Symposium 3 : Development of novel therapeutic approaches based on the pathophysiology of neurodegenerative and neurometabolic diseases>
Gene therapy for hypomyelinating leukodystrophy using AAV with artificial miRNA
Ken Inoue
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Keywords: adeno-associate virus, artificial miRNA, hypomyelination, Pelizaeus-Merzbacher disease
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2025 Volume 57 Issue 3 Pages 208-211

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[in Japanese]
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© 2025 The Japanese Society of Child Neurology
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