Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA; Department of Neurosurgery, University of Michigan, Ann Arbor, MI 48109, USA.
Department of Neurology, University of Michigan, Ann Arbor, MI 48109, USA; NeuroNetwork for Emerging Therapies, University of Michigan, Ann Arbor, MI 48109, USA.
Neurotherapeutics. 2024 Jul;21(4):e00427. doi: 10.1016/j.neurot.2024.e00427. Epub 2024 Aug 2.
Rapid advances in biological knowledge and technological innovation have greatly advanced the fields of stem cell and gene therapies to combat a broad spectrum of neurologic disorders. Researchers are currently exploring a variety of stem cell types (e.g., embryonic, progenitor, induced pluripotent) and various transplantation strategies, each with its own advantages and drawbacks. Similarly, various gene modification techniques (zinc finger, TALENs, CRISPR-Cas9) are employed with various delivery vectors to modify underlying genetic contributors to neurologic disorders. While these two individual fields continue to blaze new trails, it is the combination of these technologies which enables genetically engineered stem cells and vastly increases investigational and therapeutic opportunities. The capability to culture and expand stem cells outside the body, along with their potential to correct genetic abnormalities in patient-derived cells or enhance cells with extra gene products, unleashes the full biological potential for innovative, multifaceted approaches to treat complex neurological disorders. In this review, we provide an overview of stem cell and gene therapies in the context of neurologic disorders, highlighting recent advances and current shortcomings, and discuss prospects for future therapies in clinical settings.
生物知识和技术创新的快速进步极大地推动了干细胞和基因治疗领域的发展，以治疗广泛的神经紊乱。研究人员目前正在探索各种类型的干细胞（例如胚胎、祖细胞、诱导多能性）和各种移植策略，每种策略都有其自身的优缺点。同样，各种基因修饰技术（锌指、TALENs、CRISPR-Cas9）与各种载体一起被用于修饰神经紊乱的潜在遗传因素。虽然这两个单独的领域继续开拓新的途径，但正是这些技术的结合使得基因工程化的干细胞能够实现，并大大增加了研究和治疗的机会。在体外培养和扩增干细胞的能力，以及它们纠正患者来源细胞中遗传异常或增强带有额外基因产物的细胞的潜力，释放了创新的、多方面的治疗复杂神经紊乱的全部生物学潜力。在这篇综述中，我们在神经紊乱的背景下概述了干细胞和基因治疗，强调了最近的进展和当前的不足之处，并讨论了临床治疗的未来前景。
