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Given the approval of Luxturna and successes in transgene delivery for the RTT and FXS mouse models, this mechanism may be of value for other LoF genes implicated in ASD

Miyazono, K., Olofsson, A., Colosetti, P

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LIR has also been detected in the hypothalamus within 4 h following intravenous injection (Secher et al., 2014)

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