Delivering the Promise of Gene Therapy with Nanomedicines in Treating Central Nervous System Diseases. Issue 26 (18th July 2022)

• Record Type: Journal Article
• Authors: Luo, Meihua; Lee, Leo Kit Cheung; Peng, Bo; Choi, Chung Hang Jonathan; Tong, Wing Yin; Voelcker, Nicolas H.
• Is Part Of: Advanced science. Volume 9:Issue 26(2022)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2022-07-18
• Subjects: bio‐nanotechnology -- blood‐brain barrier -- central nervous system diseases -- gene therapy -- nanomedicine; Science -- Periodicals; 505
• DOI: 10.1002/advs.202201740
• Languages: English
• ISSNs: 2198-3844

Abstract

Abstract: Central Nervous System (CNS) diseases, such as Alzheimer's diseases (AD), Parkinson's Diseases (PD), brain tumors, Huntington's disease (HD), and stroke, still remain difficult to treat by the conventional molecular drugs. In recent years, various gene therapies have come into the spotlight as versatile therapeutics providing the potential to prevent and treat these diseases. Despite the significant progress that has undoubtedly been achieved in terms of the design and modification of genetic modulators with desired potency and minimized unwanted immune responses, the efficient and safe in vivo delivery of gene therapies still poses major translational challenges. Various non‐viral nanomedicines have been recently explored to circumvent this limitation. In this review, an overview of gene therapies for CNS diseases is provided and describes recent advances in the development of nanomedicines, including their unique characteristics, chemical modifications, bioconjugations, and the specific applications that those nanomedicines are harnessed to deliver gene therapies. Abstract : The review provides an up‐to‐date overview of various cutting‐edge gene therapies for central nervous system (CNS) diseases and describes most recent advances in the development of nanomedicines, outlining their characteristics, potential opportunities, and challenges in clinical applications. The systematic review of nanomedicine approaches provides cross‐disciplinary insights to enable the realization of advanced gene‐therapies for the treatment of CNS diseases.