Lubroth Pablo, Colasante Gaia, Lignani Gabriele
Hummingbird Ventures, London, United Kingdom.
Stem Cell and Neurogenesis Unit, Division of Neuroscience, Ospedale San Raffaele, Milan, Italy.
Front Neurosci. 2021 Feb 18;15:632522. doi: 10.3389/fnins.2021.632522. eCollection 2021.
genome editing tools, such as those based on CRISPR, have been increasingly utilized in both basic and translational neuroscience research. There are currently nine non-CNS genome editing therapies in clinical trials, and the pre-clinical pipeline of major biotechnology companies demonstrate that this number will continue to grow. Several biotechnology companies commercializing genome editing and modification technologies are developing therapies for CNS disorders with accompanying large partnering deals. In this review, the authors discuss the current genome editing and modification therapy pipeline and those in development to treat CNS disorders. The authors also discuss the technical and commercial limitations to translation of these same therapies and potential avenues to overcome these hurdles.
基因组编辑工具，如基于CRISPR的那些工具，已越来越多地用于基础和转化神经科学研究。目前有九种非中枢神经系统基因组编辑疗法正在进行临床试验，主要生物技术公司的临床前研发表明这一数字将持续增长。几家将基因组编辑和修饰技术商业化的生物技术公司正在开发针对中枢神经系统疾病的疗法，并伴有大量合作交易。在这篇综述中，作者讨论了当前基因组编辑和修饰疗法的研发情况以及正在开发的用于治疗中枢神经系统疾病的疗法。作者还讨论了这些疗法转化过程中的技术和商业限制以及克服这些障碍的潜在途径。
