Shalini Pandey, Patrick T. Bednarz, Matthias A. Oberli, Omid Veiseh
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引用次数: 0
Abstract
Over the last two decades, small activating RNAs (saRNAs) have quickly moved from discovery to clinical trials. Characterized as 20 nucleotide long, double stranded RNA, saRNAs have the unique ability to increase gene transcription at the chromatin level. This therapeutic modality has great potential as a safe and redosable alternative to gene therapy by increasing target protein expression without changing the genetic sequence. We describe the successful in vivo saRNA delivery vectors and found that similar to small interfering RNA (siRNA) and mRNA targeting tissues outside the liver works best at the end of a needle. We highlight nanoparticle vectors and RNA-conjugates, where some success has been reported for non-hepatic delivery of saRNA-aptamers.
期刊介绍：
Nano Research is a peer-reviewed, international and interdisciplinary research journal that focuses on all aspects of nanoscience and nanotechnology. It solicits submissions in various topical areas, from basic aspects of nanoscale materials to practical applications. The journal publishes articles on synthesis, characterization, and manipulation of nanomaterials; nanoscale physics, electrical transport, and quantum physics; scanning probe microscopy and spectroscopy; nanofluidics; nanosensors; nanoelectronics and molecular electronics; nano-optics, nano-optoelectronics, and nano-photonics; nanomagnetics; nanobiotechnology and nanomedicine; and nanoscale modeling and simulations. Nano Research offers readers a combination of authoritative and comprehensive Reviews, original cutting-edge research in Communication and Full Paper formats. The journal also prioritizes rapid review to ensure prompt publication.
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