KIF1A-associated neurological disorders: therapeutic opportunities and challenges
8.0
来源：
Nature
关键字：
neural coding
发布时间：
2025-11-28 04:12
摘要：
KIF1A-Associated Neurological Disorder (KAND) is a rare neurodegenerative condition caused by variants in the KIF1A gene, leading to diverse neurological symptoms. Current treatments focus on symptom management, with no cure available. The article discusses the molecular mechanisms underlying KAND and highlights emerging therapeutic strategies, including gene therapy and antisense oligonucleotide therapy, emphasizing the urgent need for targeted treatments to address the disorder's heterogeneity and improve patient outcomes.
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关键证据
KAND is a rare, progressive neurodegenerative condition with no current cure.
Emerging strategies include gene therapy and antisense oligonucleotide therapy.
The article highlights the need for targeted treatment strategies addressing the heterogeneity of KAND.
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AI评分总结
KIF1A-Associated Neurological Disorder (KAND) is a rare neurodegenerative condition caused by variants in the KIF1A gene, leading to diverse neurological symptoms. Current treatments focus on symptom management, with no cure available. The article discusses the molecular mechanisms underlying KAND and highlights emerging therapeutic strategies, including gene therapy and antisense oligonucleotide therapy, emphasizing the urgent need for targeted treatments to address the disorder's heterogeneity and improve patient outcomes.