Gene Therapy Approaches in Neurodegenerative Diseases: An Overview
DOI:
https://doi.org/10.53555/95wy1110Keywords:
Neurodegenerative diseases, Alzheimer disease, Parkinson disease, Huntington disease, ALS, Gene therapyAbstract
Neurodegenerative diseases are a major challenge in modern neuroscience. Disorders such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and Amyotrophic Lateral Sclerosis (ALS) cause serious nerve cell damage and gradual loss of body functions. Because many of these diseases are linked to faulty genes, gene therapy has gained attention as a possible way to slow or change disease progression.
In Alzheimer’s disease, gene therapy focuses on delivering genes like ApoE and BDNF to reduce harmful amyloid plaques and support nerve cell survival. Parkinson’s disease studies mainly use viral vectors to deliver genes that increase dopamine production and improve movement. Huntington’s disease treatment aims to silence the defective HTT gene using RNA-based methods and gene editing tools. In ALS, therapies target genes such as SOD1 and C9orf72 to protect motor neurons.
Modern tools like viral vectors, CRISPR-Cas9, and antisense therapies show strong potential. However, challenges such as safe delivery, side effects, immune reactions, and ethical issues remain. Overall, gene therapy offers hope for more personalized and effective treatments for neurodegenerative diseases.
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