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In the case of SMA, onasemnogene abeparvovec (AVXS-101) could deliver across the BBB and into the spinal cord without integrating into the genome of the patient [142] and was approved worldwide

Biopharm Drug Dispos 15:6573

The dopamine agonist cabergoline provides neuroprotection by activation of the glutathione system and scavenging free radicals

Repressor Element-1 Binding Transcription Factor (REST) as a possible epigenetic regulator of neurodegeneration and microRNA-based therapeutic strategies