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Rubinsztajn R, Wronska J, Chazan R

MicroRNAS (miRNAs) have been used to control exogenous transgene expression by mediating the degradation of transgene mRNAs in a tissue-specific manner and may be beneficial in RTT gene therapy approaches to limit off-target toxicity (Geisler and Fechner 2016)

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Consistently, recent evidence demonstrated that, beyond motoneuron hyperexcitability, spinal circuits in SMA undergo a maladaptive increase in inhibitory synaptic drive, resulting in a disrupted excitation/inhibition balance that further compromises motor output [35]