强迫症的基因治疗:基础研究与临床展望

Fatemeh Bamarinejad , Marzieh Shokoohi , Atefeh Bamarinejad
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引用次数: 0

摘要

背景:强迫症(OCD)是一种常见的精神疾病,以其对传统治疗的抗性而闻名。尽管有可用的药理学和心理治疗干预,但相当多的患者对这些方法没有充分的反应。最近的全基因组关联研究(GWAS)已经确定了强迫症的遗传联系,激发了人们对探索基因治疗作为替代治疗方法的兴趣。基因治疗包括纠正或替换有缺陷的基因,以针对疾病的根本原因。初步研究已显示出可喜的结果,主要集中在脑源性神经营养因子(BDNF)、催产素受体(OXTR)、SLITRK5和SLC6A4等基因上。对SHANK3和IMOOD等基因以及SLITRK5敲除小鼠等动物模型的持续探索,为潜在的治疗靶点提供了新的见解。然而,在更广泛的实施之前,必须解决道德和技术上的挑战。伦理方面的考虑包括精神疾病的多因素性质、综合治疗的必要性以及与知情同意有关的问题,特别是在患者对自己的病情缺乏了解的情况下。技术上的挑战包括安全的基因传递到大脑,潜在的脱靶效应,以及达到最佳的基因表达水平。结论强迫症的基因治疗仍处于研究和开发的早期阶段。虽然目前的研究仍处于起步阶段,但与强迫症相关的特定基因和途径的识别为未来的治疗干预提供了基础。尽管伦理和技术上的挑战仍然存在,基因工程的进步和动物模型的建立为有效的基因治疗策略的发展提供了一个充满希望的前景。随着这一领域的发展,进一步的研究是必要的,以充分认识基因治疗在治疗强迫症和相关精神疾病方面的潜力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Gene therapy for obsessive–compulsive disorder: Basic research and clinical prospects

Background

Obsessive-Compulsive Disorder (OCD) is a prevalent psychiatric condition known for its resistance to conventional treatments. Despite available pharmacological and psychotherapeutic interventions, a significant number of patients do not respond adequately to these approaches. Recent genome-wide association studies (GWAS) have identified genetic links to OCD, sparking interest in exploring gene therapy as an alternative treatment method.

Main body

Gene therapy involves correcting or replacing defective genes to target the root cause of the disorder. Preliminary studies have shown promising results, focusing on genes such as Brain Derived Neurotrophic Factor (BDNF), oxytocin receptor (OXTR), SLITRK5, and SLC6A4. Continued exploration of genes like SHANK3 and IMOOD, as well as animal models like SLITRK5 knockout mice, offer insights into potential therapeutic targets. However, ethical and technical challenges must be addressed before wider implementation can occur. Ethical considerations include the multifactorial nature of psychiatric disorders, the need for comprehensive treatment, and issues surrounding informed consent, particularly in cases where patients lack insight into their condition. Technical challenges involve safe gene delivery to the brain, potential off-target effects, and achieving optimal gene expression levels.

Conclusions

Gene therapy for OCD is still in the early stages of research and development. While current research is still in its infancy, the identification of specific genes and pathways associated with OCD offers a foundation for future therapeutic interventions. Despite the ethical and technical challenges that remain, advancements in genetic engineering and the establishment of animal models provide a hopeful outlook for the development of effective gene therapy strategies. As the field progresses, Further research is essential to fully realize the potential of gene therapy in managing OCD and related psychiatric conditions.
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