NEUROBIOLOGICAL IMPLICATIONS OF CRISPR/CAS 9 GENE EDITING IN THE TREATMENT OF NEURODEGENERATIVE DISEASES

Abstract

Gene editing with CRISPR/Cas9 technology has revolutionized the field of biomedicine, allowing precise modifications to the DNA of living cells. This breakthrough has profound implications for the treatment of various diseases, including neurodegenerative diseases, which are characterized by the progressive loss of neuronal function. Diseases such as Alzheimer’s, Parkinson’s and Amyotrophic Lateral Sclerosis (ALS) remain without a cure, and the possibility of correcting genetic mutations that cause these conditions arouses great interest. This abstract explores the neurobiological implications of using CRISPR/Cas9 technology to treat neurodegenerative diseases. This paper aims to examine the neurobiological implications of CRISPR/Cas9 gene editing in the treatment of neurodegenerative diseases, discussing recent advances, technical and ethical challenges, and the future prospects of this technology in the field of neuroscience. This study conducts a literature review on the use of CRISPR/Cas9 technology in research on neurological diseases, focusing on its applications, ethical and biological challenges, delivery strategies in the brain and impacts on neurodegeneration and neuroplasticity. The aim is to compile and critically analyze the available evidence to better understand the therapeutic potential and safety considerations involved in the use of this technology in neurological conditions. CRISPR/Cas9 technology allows the precise editing of specific genes, offering the possibility of correcting mutations that lead to the development of neurodegenerative diseases. Pre-clinical studies have shown that the application of CRISPR/Cas9 can, for example, suppress the expression of toxic proteins associated with Alzheimer’s or correct mutations in the SOD1 gene, associated with Amyotrophic Lateral Sclerosis (ALS). These advances suggest that gene editing can not only slow down the progression of the disease, but potentially reverse neurological damage. However, gene editing in the central nervous system presents significant challenges. The brain is a complex and delicate structure, where the introduction of genetic changes can have unpredictable consequences. The efficiency of delivering the CRISPR/Cas9 system to neural cells is one of the main obstacles, as is the possibility of off-target effects, where unintended edits occur in regions of the DNA, potentially leading to new genetic problems or the worsening of existing conditions. In addition, the ethical implications of gene editing in the brain are substantial. Changes to genetic material can have long-lasting impacts, not only for the patient, but also for future generations, if these changes are inheritable. Regulations and ethical consensus on the use of CRISPR/Cas9 in humans are still under development, especially in the context of neurodegenerative diseases, where the therapeutic potential is high, but the risks and uncertainties are also considerable. As a result, ongoing advances in CRISPR/Cas9 technology, including the development of more precise and less error-prone systems, are gradually making this approach safer and more viable. The combination of CRISPR/Cas9 with other techniques, such as gene therapy and the use of stem cells, is also being explored as a way of enhancing therapeutic effects and minimizing risks. Thus, the neurobiological implications of CRISPR/Cas9 gene editing in the treatment of neurodegenerative diseases are promising, with the potential to revolutionize the approach to these devastating conditions. However, technical and ethical challenges remain significant, requiring a careful and regulated approach to ensure the safety and efficacy of interventions. As the technology advances and becomes more refined, CRISPR/Cas9 could become an essential tool in the arsenal against neurodegenerative diseases, opening up new possibilities for regenerative medicine and curing previously intractable conditions.