How to package and SEND mRNA: a novel “humanized” vector system based on endogenous retroviruses  

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作  者:Kristoffer Riecken Dawid Głów Boris Fehse 

机构地区:[1]Research Dept.Cell&Gene Therapy,Dept.of Stem Cell Transplantation,University Medical Centre Hamburg-Eppendorf(UKE),Hamburg 20246,Germany

出  处:《Signal Transduction and Targeted Therapy》2021年第12期3367-3368,共2页信号转导与靶向治疗(英文)

基  金:This work has been supported by the Deutsche Forschungsgemeinschaft(DFG)within SFB841/SP2(80750187).

摘  要:A recent publication in Science describes a novel mRNA delivery system termed SEND,entirely consisting of endogenous proteins.1 These virus-like particles(VLPs),able to deliver mRNA of e.g.,Cas9,represent a new class of gene transfer vectors.The development of efficient,specific,and safe vectors for the delivery of nucleic acids and proteins remains one of the biggest challenges in gene therapy,particularly for in vivo application.The currently preferred viral vectors have several limitations,including biosafety issues,laborious and expensive manufacture,as well as the impossibility of multiple applications due to the inherent immunogenicity of viral components.In contrast,non-viral vectors are much easier to produce on a large scale,but have not yet reached gene transfer rates comparable with viral vectors.

关 键 词:ENDOGENOUS SYSTEM entirely 

分 类 号:R318[医药卫生—生物医学工程]

 

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