Folate-displaying exosome mediated cytosolic delivery of siRNA avoiding endosome trapping

Zheng, Zizhan; Li, Zhefeng; Xu, Congcong; Guo, Bin; Guo, Peixuan

doi:10.1016/j.jconrel.2019.08.021

Cited by 105 publications

(95 citation statements)

References 48 publications

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“…6); (iii) extracellular vesicles or exosomes derived from various cell types (Fig. 5f, m); 16,238,239 (iv) peptides; [240][241][242][243] (v) dendrimers; [244][245][246] (vi) RNA nanoparticles (e.g., three way-junction, 3WJ); [247][248][249][250][251][252][253][254] and (vii) inorganic nanoparticles 229,255 (Fig. 6).…”

Section: Galnac-sirna Conjugatesmentioning

confidence: 99%

“…Delivery materials derived from lipids, lipid-like materials (lipidoids), polymers, peptides, exosomes, inorganic nanoparticles, etc., have been designed and investigated. 2,8,[14][15][16][17][18][19][20][21][22] As a result, several modification patterns and delivery platforms have been employed in clinical studies. Here, the detailed evolution and advances in the modification and delivery technologies of siRNA are comprehensively summarized and discussed.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Therapeutic siRNA: state of the art

Zhong

Weng

et al. 2020

Sig Transduct Target Ther

876

705

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RNA interference (RNAi) is an ancient biological mechanism used to defend against external invasion. It theoretically can silence any disease-related genes in a sequence-specific manner, making small interfering RNA (siRNA) a promising therapeutic modality. After a two-decade journey from its discovery, two approvals of siRNA therapeutics, ONPATTRO ® (patisiran) and GIVLAARI™ (givosiran), have been achieved by Alnylam Pharmaceuticals. Reviewing the long-term pharmaceutical history of human beings, siRNA therapy currently has set up an extraordinary milestone, as it has already changed and will continue to change the treatment and management of human diseases. It can be administered quarterly, even twice-yearly, to achieve therapeutic effects, which is not the case for small molecules and antibodies. The drug development process was extremely hard, aiming to surmount complex obstacles, such as how to efficiently and safely deliver siRNAs to desired tissues and cells and how to enhance the performance of siRNAs with respect to their activity, stability, specificity and potential off-target effects. In this review, the evolution of siRNA chemical modifications and their biomedical performance are comprehensively reviewed. All clinically explored and commercialized siRNA delivery platforms, including the GalNAc (N-acetylgalactosamine)-siRNA conjugate, and their fundamental design principles are thoroughly discussed. The latest progress in siRNA therapeutic development is also summarized. This review provides a comprehensive view and roadmap for general readers working in the field.

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Section: Galnac-sirna Conjugatesmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Therapeutic siRNA: state of the art

Zhong

Weng

et al. 2020

Sig Transduct Target Ther

876

705

View full text Add to dashboard Cite

show abstract

“…Folate-modified exosomes can promote targeted delivery of siRNA and enhance the loading competence by avoiding endosome trapping. 108 A study revealed that folate-functionalized exosomes from bovine milk carrying siKRAS can reduce 54% expression of KRAS compared with the control group. 109 The high affinity and antitumor efficacy highlight the novel strategy of using folate as a cancer-targeted ligand for precisely treating cancer.…”

Section: 2mentioning

confidence: 99%

The roles of exosomes in cancer drug resistance and its therapeutic application

Dong

et al. 2020

Clinical & Translational Med

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“…8 In addition to encapsulating cytotoxic agent, exosome is also a highly promising delivery vector for gene therapy. 9,20,21 MiRNAs have extensive regulatory functions, and aberrant miRNA expression is closely with the development and progression of malignant neoplasms. Therefore, miRNA replacement therapies appear to be promising strategies for cancer treatment.…”

Section: F I G U R Ementioning

confidence: 99%