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Keywords: Adeno-associated viral vector serotype 9
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Journal Articles
Journal:
Developmental Neuroscience
Dev Neurosci (2025) 47 (2): 147–156.
Published Online: 09 May 2024
...-complementary adeno-associated viral vector serotype 9 [scAAV9]) are beyond the scope of this review and have been described previously [ 2, 10, 15 ]. Gene therapy for RTT is conceptually challenging because the gene dose sensitivity of MECP2 is complicated by the mosaic expression of endogenous MeCP2...
