Purpose: The limited efficacy of current treatments for malignant brain tumors necessitates novel therapeutic strategies. This study aimed to assess the potential of antisense oligonucleotides (ASOs) as adjuvant therapy for high-grade gliomas, focusing on their CNS penetration and clinical translation prospects. Methods: A comprehensive review of the existing literature was conducted to evaluate the implications of ASOs in neuro-oncology. Studies that investigated ASO therapy’s efficacy, CNS penetration, and safety profile were analyzed to assess its potential as a therapeutic intervention for high-grade gliomas. Results: ASOs present a promising avenue for enhancing targeted gene therapies in malignant gliomas. Their potent CNS penetration, in vivo durability, and efficient transduction offer advantages over conventional treatments. Preliminary in vivo and in vitro studies suggest ASOs as a viable adjuvant therapy for high-grade gliomas, warranting further exploration in clinical trials. Conclusions: ASOs hold significant promise as adjuvant therapy for high-grade gliomas, offering improved CNS penetration and durability compared with existing treatments. While preliminary studies are encouraging, additional research is needed to establish the safety and efficacy of ASO therapy in clinical settings. Further investigation and clinical trials are warranted to validate ASOs as a transformative approach in neuro-oncology.
目的：当前恶性脑肿瘤治疗方法的疗效有限，亟需探索新型治疗策略。本研究旨在评估反义寡核苷酸（ASOs）作为高级别胶质瘤辅助疗法的潜力，重点关注其在中枢神经系统的渗透性及临床转化前景。方法：通过系统回顾现有文献，评估ASOs在神经肿瘤学领域的应用价值。分析探讨ASO疗法疗效、中枢神经系统渗透性及安全性的相关研究，以评估其作为高级别胶质瘤治疗干预手段的潜力。结果：ASOs为增强恶性胶质瘤靶向基因治疗提供了新途径。相较于传统疗法，其强大的中枢神经系统渗透性、体内持久性及高效转导能力展现出显著优势。初步的体内外研究表明，ASOs可作为高级别胶质瘤的可行辅助疗法，值得通过临床试验进一步探索。结论：ASOs作为高级别胶质瘤辅助疗法具有重要前景，与现有治疗相比能提供更优的中枢神经系统渗透性和持久性。尽管初步研究结果令人鼓舞，仍需进一步研究以确立ASO疗法在临床环境中的安全性与有效性。有必要开展深入研究和临床试验，以验证ASOs作为神经肿瘤学领域变革性治疗方法的潜力。
Antisense Oligonucleotides for Rapid Translation of Gene Therapy in Glioblastoma
肿瘤(癌症)患者之家