Abstract: Genomic disorders affecting the central nervous system (CNS) are among the most complex and devastating conditions in human health. Moreover, these disorders, such as Rett syndrome, spinal muscular atrophy, and Fragile X syndrome, are typically caused by mutations in genes essential for neural development, synaptic function, or cellular homeostasis. Despite the genetic diversity involved, these diseases share key pathological features, including progressive neurodegeneration, disruption of neural circuits, and loss of cognitive or motor function. Meanwhile, one of the significant clinical challenges in treating CNS disorders is the limited regenerative capacity of the adult nervous system, which makes reversing disease progression extremely difficult once symptoms appear. In addition, the blood–brain barrier (BBB) restricts the passage of most systemically administered therapeutics, further complicating effective intervention. Consequently, current treatment options remain largely palliative, and effective cures remain elusive.