Neurology

Gene Therapy for Huntington’s Disease

The first gene therapy to slow Huntington’s disease progression shows promise in early trials.

2 min read

Intro

Huntington’s disease is a rare, inherited brain disorder that steadily worsens over time. In 2025, preliminary results from a clinical trial suggested that a one-time gene therapy could significantly slow its progression — marking the first therapy to directly alter the disease course.

Key Points

  • Huntington’s disease is caused by faulty repeats in the huntingtin gene, leading to toxic protein buildup in the brain.
  • Current treatments only ease symptoms; none address the underlying cause.
  • A new gene therapy slowed disease progression by ~75% in early-stage patients over three years.
  • The treatment uses a viral vector to deliver microRNA that silences the defective gene.
  • Results are preliminary (29 participants), but regulatory approval will be sought in 2026.

Background

Huntington’s disease (HD) usually begins between ages 35–55 and leads to motor problems, mood changes, and progressive cognitive decline. The disease is fatal, and treatments until now have only managed symptoms.

Causes or Mechanisms

The condition is caused by excessive DNA repeats (CAG repeats) in the huntingtin (HTT) gene. This produces a faulty protein that gradually damages neurons, especially in the striatum and cortex.

Treatment: Gene Therapy

The experimental therapy, developed by uniQure, involves:

  • Infusing a viral vector into the striatum during MRI-guided brain surgery.
  • Delivering genetic instructions for microRNA, which silences mutant huntingtin mRNA.
  • Reducing toxic protein levels in spinal fluid.
  • Slowing disease progression significantly in treated patients.

Risks / Prognosis

  • Benefits: Potentially many extra years of independence, slowed disease course.
  • Risks: Invasive surgery, still experimental, long-term effects unknown.
  • Prognosis: Not a cure, but may transform quality of life if validated in larger trials.

FAQ

Q: Is this therapy available now?
A: Not yet. Regulatory approval will be sought in 2026, depending on further trial results.

Q: Does it cure Huntington’s disease?
A: No. It slows progression but does not reverse existing brain damage.

Q: Who might benefit most?
A: People in the early stages of Huntington’s disease.

Further Reading