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).

Numerous pre-clinical and clinical studies have been conducted and are currently underway to test the efficacy of therapeutic approaches targeting some of these mechanisms with varying degrees of success.

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.

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

Explore groundbreaking therapies in development for Huntington disease, targeting its root causes and offering hope for improved patient outcomes.

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.

In the absence of disease‑modifying agents, HTT‑lowering strategies have emerged as one of the most vigorously pursued therapeutic concepts in modern neuroscience.

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...

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. Alt …

Antisense oligonucleotide programs showed that lowering huntingtin is possible in humans, but they didn’t show clear clinical benefit at scale. Those lessons informed dosing, brain‑region targeting, and endpoints in the current wave of trials.