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Although it has been suggested that further clinical trials with a patient placebo-controlled arm are required to truly confirm the efficacy of this gene therapy, this can be challenging in rare diseases like LHON

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The ionotropic receptors include NMDA (Ca 2+ influx), kainate, and AMPA (Na + Influx) receptors, while the latter one includes G protein-coupled receptors that activate the intracellular cascade (Crupi et al., 2019)

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