Introduction:
Gene therapy is rapidly becoming an innovative and promising approach to treating a variety of neurological diseases, many of which were previously considered incurable or difficult to manage. This therapeutic modality aims to correct, replace or modify defective or missing genes, offering new hope for patients suffering from neurodegenerative disorders, rare genetic diseases and other debilitating neurological conditions. (CAVASSANI NETO et al., 2024)
Advances in Gene Therapy for Neurodegenerative Diseases:
Gene therapy has shown to be a promising approach for several neurodegenerative diseases, such as Alzheimer’s, Parkinson’s, and Amyotrophic Lateral Sclerosis (ALS). In preclinical models and early clinical trials, the administration of viral vectors containing neuroprotective genes has demonstrated efficacy in slowing disease progression, preserving cognitive function, and reducing neuronal loss. For Parkinson’s disease, gene therapy has focused on modifying metabolic pathways and replacing dopamine, with promising results in clinical trials. (CAVASSANI NETO et al., 2024)
Gene Therapy for Rare Neurological Diseases:
Rare neurological diseases, many of which are genetic in origin, have benefited significantly from advances in gene therapy. Gene therapy offers the possibility of correcting the underlying genetic mutations, providing a targeted approach to treating these devastating diseases. The use of viral vectors to deliver a functional copy of the defective gene directly to hematopoietic stem cells has shown to be a promising strategy, especially in children in the early stages of the disease. (CAVASSANI NETO et al., 2024)
Gene Therapy for Psychiatric Illness and Other Neurological Disorders:
Gene therapy has also been explored as an innovative approach for the treatment of psychiatric illnesses and other neurological disorders, such as epilepsy and chronic neuropathic pain. One of the main targets of gene therapy in psychiatric illnesses has been the mesolimbic circuit, which is strongly implicated in the regulation of mood and motivation. Modulation of the expression of genes related to dopamine receptors has been investigated as a strategy to correct dopaminergic imbalances associated with disorders such as schizophrenia. (CAVASSANI NETO et al., 2024)
Challenges and Limitations in Implementing Gene Therapy:
Despite remarkable advances in the research and development of gene therapy for neurological diseases, several challenges and limitations still need to be overcome. One of the main technical challenges is the blood-brain barrier (BBB), which makes it difficult to deliver viral vectors to brain tissue. The specificity of gene expression and the immune response to viral vectors also represent significant challenges. From an ethical and regulatory perspective, gene therapy raises complex questions about the genetic modification of human cells, especially in the context of the CNS. (CAVASSANI NETO et al., 2024)
Conclusion:
Gene therapy has emerged as one of the most promising approaches for the treatment of neurological diseases, offering new hope for conditions that until recently were considered untreatable. Strategies based on the direct modification of specific genes, the delivery of neuroprotective genes, and the use of safer viral vectors represent the most promising practices in the field. However, the successful implementation of gene therapy will depend on a careful balance between scientific innovation and ethical responsibility. (CAVASSANI NETO et al., 2024)
Reference:
CAVASSANI NETO, RO et al. Advances in Gene Therapy for Neurological Diseases: Promises and Challenges. Brazilian Journal of Implantology and Health Sciences, v. 6, n. 9, p. 381-399, 2024.
