Nucleic acid delivery by ionizable nanocarriers for brain disease treatment

Brain-X Pub Date : 2023-03-26 DOI:10.1002/brx2.7
Ruru Xiong, Guixia Ling, Yuqi Zhang, Jibin Guan, Peng Zhang
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Abstract

The successful application of messenger RNA vaccines in the market has demonstrated the potential of gene therapy in treating various diseases, including infectious diseases, autoimmune disorders, brain diseases, and other cancers. However, gene therapy faces great challenges in treating brain diseases such as brain tumors, infections, and strokes because the limitations of the blood-brain barrier make it difficult for nucleic acid drugs to be delivered safely and effectively into the brain. Therefore, there is a high demand for carriers delivering nucleic acid drugs to the brain. Ionizable nanocarriers (INs) have great advantages in gene therapy due to their pH-responsive properties, which facilitate the safe and efficient delivery of targets, responsive release in the disease microenvironment, and the protection of nucleic acids from degradation. To better understand INs and their potential as therapeutic vectors for brain diseases, the present review describes their biological properties, recent progress in the field, and promising applications. In particular, the related prospects and challenges are discussed to promote the further development of INs.

Abstract Image

可电离纳米载体用于脑疾病治疗的核酸递送
信使核糖核酸疫苗在市场上的成功应用证明了基因治疗在治疗各种疾病方面的潜力,包括传染病、自身免疫性疾病、脑疾病和其他癌症。然而,基因治疗在治疗脑肿瘤、感染和中风等脑部疾病方面面临巨大挑战,因为血脑屏障的局限性使核酸药物难以安全有效地输送到大脑中。因此,对将核酸药物输送到大脑的载体有很高的需求。可电离纳米载体(in)由于其pH响应特性,在基因治疗中具有巨大优势,有助于安全有效地递送靶标,在疾病微环境中响应释放,并保护核酸免受降解。为了更好地了解in及其作为脑疾病治疗载体的潜力,本文综述了其生物学特性、该领域的最新进展和有前景的应用。特别是,讨论了相关的前景和挑战,以促进In的进一步发展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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