Target intelligence / Profile preview

Kinesin-like protein KIF1A (KIF1A)

Target
KIF1A
Molecular classification
Microtubule-based motor protein, Kinesin superfamily (kinesin-3 family), Enzyme (ATPase)
01

Overview

Kinesin-like protein KIF1A is a neuron-specific, microtubule-based motor protein encoded by the KIF1A gene in humans[1]. It belongs to the kinesin-3 subfamily and primarily mediates long-distance anterograde axonal transport of membranous cargoes, including synaptic vesicle precursors and dense core vesicles, by hydrolyzing ATP to generate mechanical force and “walk” along microtubules[1][3]. KIF1A is essential for neuronal survival, synaptic transmission, brain development, and higher cognitive function[1]. Disruption or mutation of KIF1A leads to severe neurodevelopmental and neurodegenerative disorders, collectively known as KIF1A-associated neurological disorders (KAND), characterized by spasticity, intellectual disability, and peripheral neuropathy[2][4][5]. Mechanistically, KIF1A is distinguished by high processivity and rapid movement powered by structural features such as its motor domain and the lysine-rich K-loop, allowing efficient delivery of cargo throughout the neuron[3][5]. While KIF1A is a recognized therapeutic target due to its role in neuronal health and disease, clinically relevant modulators have not yet been described in the literature[5].

Other names
KIF1AATSVC2orf20hUnc-104UNC104Axonal transporter of synaptic vesiclesMicrotubule-based motor KIF1AUnc-104- and KIF1A-related proteinHSN2CMRD9NESCAVSSPG30SPG30ASPG30B
02

Biological functions

Anterograde transport of synaptic vesicle precursors and dense core vesiclesNeuronal survivalSynaptic transmission and plasticityAxonal transport of organelles and protein complexesRegulation of learning and memory
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Disease associations

Neurodegenerative diseaseHereditary sensory neuropathy (HSN2C)Mental retardation type 9 (MRD9)Hereditary spastic paraplegia (SPG30/30A/30B, HSP)KIF1A-associated neurological disorder (KAND)
04

Safety considerations

Mutations can cause loss-of-function or gain-of-function effects, leading to neurodevelopmental/degenerative disorders (e.g., spastic paraplegia, intellectual disability)[2][5].Disruption of KIF1A function can impair synaptic transmission, axonal transport, or cause abnormal vesicle accumulation—both impairments and hyperactivity are detrimental to neuronal health and function[2][4][6].No specific small molecules or drugs with defined safety profiles for KIF1A modulation in humans are reported in search results as of the current date.

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