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<p>An abnormal amplification of the CAG trinucleotide repeat in the huntingtin (<i>HTT</i>) gene causes Huntington’s disease (HD), a devastating neurological illness. The striatum and cortex are primarily affected by the gradual neuronal dysfunction and loss caused by the mutant huntingtin (mHTT) protein. Although there have been notable improvements in symptomatic management, curative treatments remain unavailable. A promising treatment option that offers specifically designed interventions to decrease or eradicate mutant <i>HTT</i> expression is gene therapy. RNA-targeted treatments (antisense oligonucleotides, RNA interference), DNA editing methods (Clustered Regularly Interspaced Short Palindromic Repeats/caspase 9, zinc finger nucleases, Transcription Activator-like Effector Nuclease), and advanced delivery systems (viral and non-viral vectors, lipid nanoparticles, exosomes) are just a few of the many approaches that are presently being researched. AMT-130 and tominersen clinical trials provide crucial information about the viability, security, and effectiveness of gene therapy for HD. It is hoped that developments in genome editing and delivery methods would make gene therapy a viable treatment for HD. However, challenges remain, including immunological responses, blood-brain barrier penetration, and off-target consequences. This review delves into the latest developments in HD gene therapy, highlighting new approaches, obstacles, and potential future paths to a permanent cure.</p>

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Gene therapy for huntington’s disease: advances, challenges, and future perspectives

  • 期刊论文

  • 发表日期：

    2026年2月21日

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  • Riya Kaushal,
  • Mohit Yadav,
  • Sourabh Kosey,
  • Madhaw Dwivedi

摘要

An abnormal amplification of the CAG trinucleotide repeat in the huntingtin (HTT) gene causes Huntington’s disease (HD), a devastating neurological illness. The striatum and cortex are primarily affected by the gradual neuronal dysfunction and loss caused by the mutant huntingtin (mHTT) protein. Although there have been notable improvements in symptomatic management, curative treatments remain unavailable. A promising treatment option that offers specifically designed interventions to decrease or eradicate mutant HTT expression is gene therapy. RNA-targeted treatments (antisense oligonucleotides, RNA interference), DNA editing methods (Clustered Regularly Interspaced Short Palindromic Repeats/caspase 9, zinc finger nucleases, Transcription Activator-like Effector Nuclease), and advanced delivery systems (viral and non-viral vectors, lipid nanoparticles, exosomes) are just a few of the many approaches that are presently being researched. AMT-130 and tominersen clinical trials provide crucial information about the viability, security, and effectiveness of gene therapy for HD. It is hoped that developments in genome editing and delivery methods would make gene therapy a viable treatment for HD. However, challenges remain, including immunological responses, blood-brain barrier penetration, and off-target consequences. This review delves into the latest developments in HD gene therapy, highlighting new approaches, obstacles, and potential future paths to a permanent cure.