-然而,所有以mRNA为基础的药物都必须进入细胞质,外源mRNA才能在细胞质中被翻译,这与它们最终的作用机制无关。这是一个挑战,因为高分子量的负离子分子不能自己跨越细胞双层膜。 -为了应对这些挑战,科学家们开发了非病毒传递系统,该系统可以保护mRNA免受RNAse介导的降解,帮助mRNA进入靶细胞,并减少传递到脱靶细胞。 -...
图8,基于LNP的mRNA治疗 Ref Kim, J., Eygeris, Y., Ryals, R.C. et al. Strategies for non-viral vectors targeting organs beyond the liver. Nat. Nanotechnol. (2023). Abramson, A. et al. An ingestible self-orienting system for oral ...
An orthogonal array optimization of lipid-like nanoparticles for mRNA delivery in vivo. Nano Lett. 15, 8099–8107 (2015). Article CAS Google Scholar DeRosa, F. et al. Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system. Gene Ther. 23, 699–707 (2016...
For systemic delivery of DNA, both lipid-based vectors and polymer-based vectors have been intensively investigated in experimental animals and in clinical trials. The potential of mRNA for therapeutic protein expressionin vivohas been investigated as an alternative to DNA-based gene therapy owing to...
Fig. 3. mRNA vaccine delivery systems applicable for COVID-19 vaccine development. To develop an effective LNP vector for delivery of COVID-19 mRNA vaccine, two critical factors should be taken into consideration. The first factor is choosing an appropriate cationic or ionizable lipid [16]. Ev...
[12,99]. Non-viral delivery platforms are advancing with great speed towards the clinic, tailored to treat a range of different diseases such as COVID-19, genetic disorders and malignant neoplasms (by delivery of therapeutic oligonucleotides and aptamers such as RNA, DNA, siRNA, mRNA and ...
$%^&*AU2018203310B220200514.pdf### ABSTRACT Compositions comprising mRNA formulated for pulmonary administration and related methods for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue. The compositions and methods may be used to prevent or ameliorate the symptoms of ...
Several liposome-encapsulated DNA (NCT01502358) or mRNA (NCT03382405) in vivo delivery systems have entered into clinical trials, and they are also frequently used to deliver lncRNAs in preclinical experiments in vitro and in vivo110, 111, 112. Liposomes can be cationic or cationic-anionic ...
As the main form of biological communication, EV delivery of ncRNAs in liver cancer has been thoroughly reviewed. Herein, as the third molecular regulatory tool, we summarize the functions of these exosomes and MV-containing biomolecules in the regulation of progression and therapeutic efficacy in ...
Finally, lncRNAs hold promise for development of novel tailored therapeutic approaches. The proposed weaponry to target lncRNAs would rely on the delivery of RNA-based drugs, mostlysmall interfering RNAs, to silence the abnormal functioning of these particles. As for most of thegenetictherapies, sever...