中国神经再生研究(英文版) ›› 2026, Vol. 21 ›› Issue (8): 3547-3548.doi: 10.4103/NRR.NRR-D-25-00766

• 观点:神经损伤修复保护与再生 • 上一篇    下一篇

纳米粒子辅助基因编辑治疗中枢神经系统基因组疾病

  

  • 出版日期:2026-08-18 发布日期:2026-04-25

Nanoparticle-assisted gene editing for genomic disorders in the central nervous system

Hyeon-Yeol Cho, Jeong-Woo Choi*   

  1. Department of Bio & Fermentation Convergence Technology, Kookmin University, Seoul, South Korea (Cho HY)
    Department of Chemical and Biomolecular Engineering, Sogang University, Seoul, South Korea (Choi JW)
  • Online:2026-08-18 Published:2026-04-25
  • Contact: Jeong-Woo Choi, PhD, DEng, DBA, jwchoi@sogang.ac.kr.
  • Supported by:
    JWC acknowledges the financial support from the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) (RS-2024-00344633). HYC acknowledges the financial support from the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) (RS-2023-00211360) and Biomaterials Specialized Graduate Program through the Korea Environmental Industry & Technology Institute (KEITI) funded by the Ministry of Environment (MOE).

摘要: https://orcid.org/0000-0003-0100-0582 (Jeong-Woo Choi)
https://orcid.org/0000-0003-1897-1166 (Hyeon-Yeol Cho)

Abstract: Genomic disorders affecting the central nervous system (CNS) are among the most complex and devastating conditions in human health. Moreover, these disorders, such as Rett syndrome, spinal muscular atrophy, and Fragile X syndrome, are typically caused by mutations in genes essential for neural development, synaptic function, or cellular homeostasis. Despite the genetic diversity involved, these diseases share key pathological features, including progressive neurodegeneration, disruption of neural circuits, and loss of cognitive or motor function. Meanwhile, one of the significant clinical challenges in treating CNS disorders is the limited regenerative capacity of the adult nervous system, which makes reversing disease progression extremely difficult once symptoms appear. In addition, the blood–brain barrier (BBB) restricts the passage of most systemically administered therapeutics, further complicating effective intervention. Consequently, current treatment options remain largely palliative, and effective cures remain elusive.