中国神经再生研究(英文版) ›› 2022, Vol. 17 ›› Issue (8): 1717-1725.doi: 10.4103/1673-5374.332129

• 综述:脑损伤修复保护与再生 • 上一篇    下一篇

基于siRNA 的脑出血疗法:药物输送系统的挑战和进展

  

  • 出版日期:2022-08-15 发布日期:2022-01-21

Small interfering RNAs based therapies for intracerebral hemorrhage: challenges and progress in drug delivery systems

Daniyah A. Almarghalani1, 2, Sai H.S. Boddu3, Mohammad Ali2, Akhila Kondaka2, Devin Ta2, Rayyan A. Shah2, Zahoor A. Shah2, *   

  1. 1Department of Pharmacology and Experimental Therapeutics, 2Department of Medicinal and Biological Chemistry, College of Pharmacy and Pharmaceutical Sciences, University of Toledo, Toledo, OH, USA; 3Department of Pharmaceutical Sciences, College of Pharmacy and Health Sciences, Ajman University, Ajman, United Arab Emirates
  • Online:2022-08-15 Published:2022-01-21
  • Contact: Zahoor A. Shah, PhD, zahoor.shah@utoledo.edu.
  • Supported by:
    A Scholarship supported Daniyah Almarghalani from Taif University, Saudi Arabia Cultural Mission. The work was partly supported by the grants from American Heart Association #17AIREA33700076/ZAS/2017 and the National Institute of Neurological Disorders and Stroke of the National Institutes of Health #R01NS112642 to ZAS.

摘要: https://orcid.org/0000-0001-8977-1618 (Zahoor A. Shah)

Abstract: Intracerebral hemorrhage (ICH) is a subtype of stroke associated with higher rates of mortality. Currently, no effective drug treatment is available for ICH. The molecular pathways following ICH are complicated and diverse. Nucleic acid therapeutics such as gene knockdown by small interfering RNAs (siRNAs) have been developed in recent years to modulate ICH’s destructive pathways and mitigate its outcomes. However, siRNAs delivery to the central nervous system is challenging and faces many roadblocks. Existing barriers to systemic delivery of siRNA limit the use of naked siRNA; therefore, siRNA-vectors developed to protect and deliver these therapies into the specific-target areas of the brain, or cell types seem quite promising. Efficient delivery of siRNA via nanoparticles emerged as a viable and effective alternative therapeutic tool for central nervous system-related diseases. This review discusses the obstacles to siRNA delivery, including the advantages and disadvantages of viral and nonviral vectors. Additionally, we provide a comprehensive overview of recent progress in nanotherapeutics areas, primarily focusing on the delivery system of siRNA for ICH treatment.

Key words: intracerebral hemorrhage, lipid-based nanoparticle, nanoparticles, nanotechnology, nonviral vectors, peptide-mediated nanoparticle, polymer-based nanoparticle, siRNA therapeutics, siRNA-barriers, viral vectors