Gene therapy approaches for neurodegenerative diseases

🕒 Approximate reading time: 5 minutes

Neurodegenerative diseases, characterised by progressive neuronal loss and cognitive decline, remain a significant medical challenge. However, the frontier of gene therapy is offering novel and exciting therapeutic options. This article will delve into the current state of gene therapy in combating neurodegenerative diseases.

Understanding the Basics

Gene Therapy Defined: Gene therapy is a technique that modifies genes within an individual's cells to treat or prevent diseases.

Targeted Approach: Gene therapies focus on specific genes linked to particular conditions, enabling precise treatment methods.

Types of Gene Therapies

Gene Addition: Introducing a new, functional gene to compensate for a faulty one.

Gene Silencing: Suppressing the activity of a malfunctioning gene.

Gene Editing: Directly altering the faulty DNA sequence to its normal state using tools like CRISPR.

Gene Therapies in Practice

Parkinson's Disease: Trials have explored delivering genes that can either increase dopamine production or counteract its loss.

Huntington's Disease: Research is focused on silencing the mutant huntingtin gene causing the disease.

Alzheimer’s Disease: Efforts centre around genes influencing amyloid-beta plaques or tau protein accumulation.

Delivery Mechanisms

Viral Vectors: Using viruses, which are adept at entering human cells, to deliver the desired genetic material. Adeno-associated viruses (AAV) are commonly used due to their low risk of causing diseases.

Nanoparticles: Tiny particles can encapsulate genetic material, ensuring it gets into the target cells.

Challenges and Limitations

Safety Concerns: Manipulating genes can sometimes have unforeseen side effects or cause adverse reactions.

Efficiency: Ensuring that the therapy reaches enough cells to have a therapeutic effect.

Regulation and Ethical Concerns: The potential for gene editing, especially in germline cells, raises ethical dilemmas.


Gene therapy stands as a beacon of hope for countless individuals affected by neurodegenerative diseases. While still in its infancy and facing challenges, the strides made in this arena are undeniably groundbreaking. With further research and technological advances, gene therapy might significantly alter the treatment landscape of neurodegenerative conditions.