Abstract：
In a recent issue of Nature, Chen and colleagues1 reveal the potential for antisense oligonucleotides (ASOs) to rescue the neuropathological mechanisms underlying Timothy syndrome (TS) using three-dimensional neuronal models. Combining in vitro and in vivo approaches, the authors present a strategy to translate disease biology findings into potential therapeutics.
摘要：
在最近一期的《自然》杂志上，Chen和同事1揭示了反义寡核苷酸（ASO）使用三维神经元模型挽救蒂莫西综合征（TS）潜在的神经病理学机制的潜力。结合体外和体内方法，作者提出了一种将疾病生物学发现转化为潜在治疗方法的策略.
