Use of Antisense Oligonucleotides as Therapy for Huntington's Disease

Abstract

Introduction: Huntington's disease is a neurodegenerative disorder characterized by the progressive degeneration of nerve cells in the brain, and whose current treatment focuses on the control of symptoms, which is why in recent years several therapies have been evaluated, including Antisense oligonucleotides a promising strategy, thanks to their ability to modulate the expression of the mutated huntingtin protein. Methods: a bibliographic search was carried out using the PRISMA methodology in databases such as PubMed, limited to documents in English published during the last 5 years, using keywords such as "Huntington's Disease", "Antisense Oligonucleotides", "Treatment" “Tominersen”, selecting only documents such as original articles, bibliographic reviews and clinical trial reports related to the use of Antisense Oligonucleotides as therapy for Huntington's Disease. Results: 150 articles were collected and analyzed, of which 30 documents were excluded due to their age and 40 due to lack of access and quality of information, leaving a total of 80 articles to which inclusion criteria were applied, selecting 25 articles for completion. of this literature review on the use of antisense oligonucleotides in Huntington's disease Conclusion: antisense oligonucleotides show great therapeutic potential for Huntington's disease by directly attacking the underlying cause of the disease, mutated huntingtin. However, significant challenges still remain, which is why better research is needed to ensure efficacy. and safety of this therapy in the long term