Therapeutic agents designed to lower huntingtin levels, currently in development, target HTT DNA through methods such as modulating gene transcription or directly modifying the HTT gene (genome editing).

Presently, therapeutic interventions capable of altering the trajectory of HD are lacking, while medications for abnormal movement and psychiatric symptoms are limited.

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.

Although new treatment options for Huntington's disease are urgently needed, the recent AMT-130 gene therapy trial offers a promising source of hope. Current treatment options focus primarily on symptom management and supportive measures only. AMT-130 is a one-time gene therapy targeting mutant HTT.

Scientists have been striving to present the latest research on gene therapy for Huntington's disease. In this review, we will examine various gene therapy techniques that involve...

Recent Findings HD is living in an era of target-specific drug development with emphasis on the mechanisms related to mutant Huntingtin (HTT) protein. Examples include antisense oligonucleotides (ASO), splicing modifiers and microRNA molecules that aim to reduce the levels of mutant HTT protein.

This exhaustive review discusses different disease-modifying treatments, their clinical progress, and pharmaceutical developments for Huntington's therapy. Moreover, it will guide to develop effective targeted therapeutics for HD.

Additionally, current management approaches and emerging therapeutic strategies—such as RNA interference, antisense oligonucleotides (ASOs), peptide inhibitors, and CRISPR/Cas9 gene editing—are discussed.

immunological responses, blood-brain barrier penetration, and off-target consequences. This review delves into the latest developments in HD gene therapy, Keywords Huntington’s disease · Gene therapy · RNA interference · CRISPR/Cas9 · Zinc finger proteins · Antisense oligonucleotides

We comprehensively review the mechanisms of CRISPR-based therapeutic strategies, including excision of expanded repeats, allele-specific targeting, and epigenome editing, highlighting their efficacy in preclinical studies using animal models and human iPSCs.