Advancements in Genetic Engineering
Open Access

Treatment of Neurodegenerative Diseases using Gene therapy

Sara Stanley^{* *}Correspondence: Sara Stanley, Department of Biochemistry and Molecular Biology, University Sultan Moulay Slimane, Khouribga, Morocco, Email:

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Description

Neurodegenerative diseases are a class of debilitating disorders that progressively affect the nervous system, leading to the gradual degeneration and loss of function of nerve cells. These diseases, which include Alzheimer's disease, Parkinson's disease, Huntington's disease, and Amyotrophic Lateral Sclerosis (ALS), have long posed significant challenges to the medical community due to their complex and largely incurable nature. However, in recent years, gene therapy has emerged as a promising avenue for revolutionizing the treatment of neurodegenerative diseases.

Neurodegenerative diseases are characterized by the progressive deterioration of specific populations of neurons in the brain and spinal cord. This degeneration typically results in a wide range of cognitive, motor, and sensory impairments. While the exact causes of these diseases vary, many of them are linked to the accumulation of abnormal proteins, genetic mutations, or a combination of both.

Treatment or prevent diseases of gene therapy

Gene therapy is a cutting-edge medical approach that aims to treat or prevent diseases by modifying or replacing a person's faulty genes. In the context of neurodegenerative diseases, gene therapy holds great promise for several reasons:

Targeted correction: Gene therapy allows for precise targeting of the genetic defects responsible for neurodegenerative diseases. By delivering therapeutic genes directly to affected cells, researchers can potentially halt or even reverse the disease progression.

Reducing toxic proteins: Many neurodegenerative diseases involve the accumulation of toxic proteins within nerve cells. Gene therapy strategies can be designed to reduce the production of these toxic proteins or enhance the clearance mechanisms, thereby slowing down the disease progression.

Neuroprotection: Some gene therapies are designed to protect and promote the survival of vulnerable neurons. This can help in preserving cognitive and motor functions for a longer period, significantly improving the quality of life for patients.

Personalized medicine: Gene therapy can be tailored to an individual's genetic profile, making it a form of personalized medicine. This approach has the potential to be more effective and have fewer side effects compared to one-size-fits-all treatments.

Gene therapy approaches for neurodegenerative diseases

Several gene therapy strategies are being explored for neurodegenerative diseases:

Gene replacement: In cases where a faulty gene is responsible for the disease, gene therapy can introduce a healthy copy of the gene into affected cells. For example, in Spinal Muscular Atrophy (SMA), a gene therapy called Zolgensma has been approved, which provides a functional copy of the SMN1 gene.

Ribonucleic acid (RNA) interference: RNA interference (RNAi) therapies can silence genes that produce harmful proteins. This approach is being studied for conditions like Huntington's disease, where the mutant huntingtin protein can be targeted.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-based therapies: The revolutionary CRISPR-Cas9 gene-editing technology holds promise for directly correcting genetic mutations responsible for neurodegenerative diseases. Researchers are working on refining the precision of this technique for safe and effective application.

Conclusion

Gene therapy represents a groundbreaking frontier in the treatment of neurodegenerative diseases. While challenges remain, the progress made in recent years is promising. With continued research, advancements in delivery methods, and rigorous safety assessments, gene therapy has the potential to offer hope to millions of individuals and families affected by these devastating diseases. As our understanding of genetics and gene therapy techniques evolves, the day when neurodegenerative diseases become manageable or even curable draws closer.