KIF1A-associated neurological disorder (KAND) is an extremely rare neurodegenerative condition, frequently resulting in severe outcomes, characterized by a diverse range of symptoms linked to predominantly heterozygous de novo missense mutations in the KIF1A gene. The phenotypic characteristics encompass brain and optic nerve atrophy, resistant epilepsy, cognitive deficits, spasticity, and neuropathy. Antisense oligonucleotide therapies offer a hopeful avenue for personalized treatment options in such ultrarare diseases. KIF1A is a neuron-specific member of the kinesin-3 family and acts as a motor protein involved in the anterograde transport of cargo along axonal microtubules. Multiple de novo mutations have been identified in the KIF1A motor domain, resulting in a loss of motility or functionality of the motor protein. Here, we will study the de novo pathogenic c.946C>T variant in KIF1A (p. R316W), which was shown to act as a dominant negative variant and used in this study as a prototype for ASO/s (antisense aligonucleotide/s) development and personalised treatment