Department of Neurology, Applied Neurobiology Group, University Medical Center of the Heinrich Heine University Düsseldorf Düsseldorf, Germany.
Front Mol Neurosci. 2011 Sep 23;4:25. doi: 10.3389/fnmol.2011.00025. eCollection 2011.
This mini-review focuses on a knockdown technology called deoxyribozymes, which has rarely been utilized in the field of neurobiology/neuroscience. Deoxyribozymes are catalytic DNA molecules, which are also entitled DNA enzyme or DNAzyme. This mini-review presents a description of their development, structure, function, and therapeutic application. In addition, information on siRNA, ribozymes, and antisense are given. Further information on two deoxyribozymes against c-Jun and xylosyltransferase (XT) mRNA are summarized of which the first is important to influence many neurological disorders and the last potentially treats spinal cord injuries (SCIs). In particular, insults to the central nervous system (CNS) such as SCI generate an inhibitory environment (lesion scar) at the injury site that prevents the endogenous and therapy-induced axonal regeneration and thereby limits repair strategies. Presently, there are no treatments available. Hence, deoxyribozymes provide an opportunity for new therapeutics that alter the inhibitory nature of the lesion scar and thus promote axonal growth in the injured spinal cord. When used cautiously and within the limits of its ability the deoxyribozyme technology holds promise to become a major contributing factor in repair strategies of the CNS.
这篇小型综述专注于一种称为脱氧核酶的敲低技术，该技术在神经生物学/神经科学领域很少被应用。脱氧核酶是催化性 DNA 分子，也被称为 DNA 酶或 DNAzyme。本小型综述介绍了它们的发展、结构、功能和治疗应用。此外，还提供了有关 siRNA、核酶和反义寡核苷酸的信息。对两种针对 c-Jun 和木糖基转移酶 (XT) mRNA 的脱氧核酶的进一步信息进行了总结，其中第一种对影响许多神经疾病很重要，而最后一种可能治疗脊髓损伤 (SCI)。特别是，中枢神经系统 (CNS) 的损伤，如 SCI，会在损伤部位产生抑制性环境（损伤瘢痕），阻止内源性和治疗诱导的轴突再生，从而限制修复策略。目前尚无有效的治疗方法。因此，脱氧核酶为改变损伤瘢痕的抑制性质并促进损伤脊髓中轴突生长的新疗法提供了机会。当脱氧核酶技术谨慎地应用于其能力范围内时，有望成为中枢神经系统修复策略的主要贡献因素。
