Non‐viral nucleic acid delivery to the central nervous system and brain tumors

Wang, Shanshan; Huang, Rongqin

doi:10.1002/jgm.3091

Cited by 15 publications

(10 citation statements)

References 96 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…For efficient gene delivery, it is essential to load gene drugs into a delivery vector, which is divided into viral vector and non-viral vector. The further clinical application of viral vectors is limited due to the biological safety problems such as cytotoxicity, high immunogenicity and potential carcinogenicity [10 , 11] . At present, non-viral gene delivery vectors such as cationic polymers [12] , cationic polysaccharides [13] , protamine [14] , cationic lipids [15] and cationic surfactants [16] have become the focus of gene therapy research due to low toxicity, low immunogenicity, etc .…”

Section: Introductionmentioning

confidence: 99%

Anti-EpCAM functionalized graphene oxide vector for tumor targeted siRNA delivery and cancer therapy

Chen

Zhang

Wang

et al. 2021

Asian Journal of Pharmaceutical Sciences

View full text Add to dashboard Cite

Graphene oxide (GO) has emerged as a potential drug delivery vector. For siRNA delivery, GO should be modified to endow it with gene delivery ability and targeting effect. However, the cationic materials used previously usually had greater toxicity. In this study, GO was modified with a non-toxicity cationic material (chitosan) and a tumor specific monoclonal antibody (anti-EpCAM) for the delivery of survivin-siRNA (GCE/siRNA). And the vector (GCE) prepared was proved with excellent biosafety and tumor targeting effect. The GCE exhibited superior performance in loading siRNA, maintained stability in different solutions and showed excellent protection effect for survivin-siRNA in vitro . The gene silencing results in vitro showed that the mRNA level and protein level were down-regulated by 48.24% ± 2.50% and 44.12% ± 3.03%, respectively, which was equal with positive control ( P > 0.05). It was also demonstrated that GCE/siRNA had a strong antitumor effect in vitro , which was attributed to the efficient antiproliferation, and migration and invasion inhibition effect of GCE/siRNA. The results in vivo indicated that GCE could accumulate siRNA in tumor tissues. The tumor inhibition rate of GCE/siRNA 54.74% ± 5.51% was significantly higher than control 4.87% ± 8.49%. Moreover, GCE/siRNA showed no toxicity for blood and main organs, suggesting that it is a biosafety carrier for gene delivery. Taken together, this study provides a novel design strategy for gene delivery system and siRNA formulation.

show abstract

Section: Introductionmentioning

confidence: 99%

Anti-EpCAM functionalized graphene oxide vector for tumor targeted siRNA delivery and cancer therapy

Chen

Zhang

Wang

et al. 2021

Asian Journal of Pharmaceutical Sciences

View full text Add to dashboard Cite

show abstract

“…Local intraparenchymal injections are the most common delivery method for circumventing the BBB, but some AAV serotypes have been shown to cross the BBB after systemic delivery (Choudhury et al, 2016b;Chan et al, 2017;Hudry et al, 2018). Non-viral vehicles are generally less efficient than viral vectors, but the development of non-viral delivery methods for the CNS is an intense field of research and may open up new possibilities for treatment in the near future (Wang and Huang, 2019). The immunogenicity induced by genome editing tools is another topic of concern due to potential inflammatory responses (Shim et al, 2017).…”

Section: Future Perspectives In Genome Editing For Cns Disordersmentioning

confidence: 99%

Genome Editing for CNS Disorders

Duarte

Déglon

2020

Front. Neurosci.

View full text Add to dashboard Cite

Central nervous system (CNS) disorders have a social and economic burden on modern societies, and the development of effective therapies is urgently required. Gene editing may prevent or cure a disease by inducing genetic changes at endogenous loci. Genome editing includes not only the insertion, deletion or replacement of nucleotides, but also the modulation of gene expression and epigenetic editing. Emerging technologies based on ZFs, TALEs, and CRISPR/Cas systems have extended the boundaries of genome manipulation and promoted genome editing approaches to the level of promising strategies for counteracting genetic diseases. The parallel development of efficient delivery systems has also increased our access to the CNS. In this review, we describe the various tools available for genome editing and summarize in vivo preclinical studies of CNS genome editing, whilst considering current limitations and alternative approaches to overcome some bottlenecks.

show abstract

“…In the case of inborn metabolism mutations of one gene that affect the brain, such as mucopolysaccharidoses or Canavan disease, the approach normally consists of the delivery of a functional copy of the gene to restore the normal phenothype [146][147][148]. However, in the case of brain-acquired diseases, where more than one gene can be affected, such as brain cancer, Alzheimer's, or Parkinson's diseases, the genetic approach is more challenging, since the molecular basis of those disorders are still not understood [148][149][150]. BBB hampers the entry of gene expression vectors into the brain; consequently, gene treatments must be given after an invasive craniotomy, which in many cases jeopardizes the acceptance of patients enrolled in clinical trials due to the cumbersome approach and related side effects that increase the after-care cost as a consequence of additional hospital visits [151].…”

Section: General Conceptsmentioning

confidence: 99%

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues

et al. 2020

View full text Add to dashboard Cite

Non-viral vectors have emerged as a promising alternative to viral gene delivery systems due to their safer profile. Among non-viral vectors, recently, niosomes have shown favorable properties for gene delivery, including low toxicity, high stability, and easy production. The three main components of niosome formulations include a cationic lipid that is responsible for the electrostatic interactions with the negatively charged genetic material, a non-ionic surfactant that enhances the long-term stability of the niosome, and a helper component that can be added to improve its physicochemical properties and biological performance. This review is aimed at providing recent information about niosome-based non-viral vectors for gene delivery purposes. Specially, we will discuss the composition, preparation methods, physicochemical properties, and biological evaluation of niosomes and corresponding nioplexes that result from the addition of the genetic material onto their cationic surface. Next, we will focus on the in situ application of such niosomes to deliver the genetic material into immune-privileged tissues such as the brain cortex and the retina. Finally, as future perspectives, non-invasive administration routes and different targeting strategies will be discussed.

show abstract

Non‐viral nucleic acid delivery to the central nervous system and brain tumors

Cited by 15 publications

References 96 publications

Anti-EpCAM functionalized graphene oxide vector for tumor targeted siRNA delivery and cancer therapy

Anti-EpCAM functionalized graphene oxide vector for tumor targeted siRNA delivery and cancer therapy

Genome Editing for CNS Disorders

Niosome-Based Approach for In Situ Gene Delivery to Retina and Brain Cortex as Immune-Privileged Tissues

Contact Info

Product

Resources

About