The Whitehead siRNA (short interfering RNA) Selection Web Server (http://jura.wi.mit.edu/bioc/siRNA) automates the design of short oligonucleotides that can specifically 'knock down' expression of target genes. These short sequences are about 21 nt in length, and when synthesized as double stranded RNA and introduced into cell culture, can reduce or eliminate the function of the target gene. De...

This article reviews the pulmonary route of administration, aerosol delivery devices, characterization of pulmonary drug delivery systems, and discusses the rationale for inhaled delivery of siRNA. Diseases with known protein malfunctions may be mitigated through the use of siRNA therapeutics. The inhalation route of administration provides local delivery of siRNA therapeutics for the treatment...

The development of functional lipid nanoparticles (LNPs) is one the major challenges in field drug delivery systems (DDS). Recently, LNP-based RNA systems, namely, RNA-loaded LNPs have attracted attention for therapy. In particular, mRNA-loaded LNP vaccines were approved to prevent COVID-19, thereby leading paradigm shift toward next-generation nanomedicines. For nanomedicines, size a significa...

Bmi1 gene overexpression is found in various human tumors and has been shown as a potential target for gene treatment. However, siRNA-based treatments targeting Bmi1 gene have been restricted to limited delivery, low bioavailability and hence relatively reduced efficacy. To overcome these barriers, we developed a folate receptor targeted co-delivery system folate-doxorubicin/Bmi1 siRNA liposome...

RNA interference has become widely used as an experimental tool to study gene function. In addition, small interfering RNA (siRNA) may have great potential for the treatment of diseases. Recently, it was shown that siRNA can be used to mediate gene silencing in mouse models. Locally administered siRNAs entered the first clinical trials, but strategies for successful systemic delivery of siRNA a...

Nanoparticle-mediated small interfering RNA (siRNA) delivery is a promising therapeutic strategy in various cancers. However, it is difficult to deliver degradative siRNA to tumor tissue, and thus a safe and efficient vector for siRNA delivery is essential for cancer therapy. In this study, poly(ethylene glycol)-modified chitosan (PEG-CS) was synthesized successfully for delivering nucleic acid...

Introduction Recombinant Dicer generated siRNA (d-siRNA) has been demonstrated to be as effective as chemically synthesized siRNA (cs-siRNA) at targeted gene knock-down.1 However, many scientists have presumed that d-siRNA will produce significant offtarget gene suppression since there are multiple species of siRNA present in a d-siRNA pool. Yet, no group until recently has investigated whether...

Matrix metalloproteinase-2 (MMP-2) is constitutively expressed in vascular smooth muscle cells (VSMCs). Using small interfering RNA (siRNA), we evaluated the effect of MMP-2 inhibition in VSMCs in vitro and ex vivo. Rabbit VSMCs were transfected in vitro with 50 nmol/l MMP-2 siRNA or scramble siRNA. Flow cytometry and confocal microscopy showed cellular uptake of siRNA in approximately 80% of V...

Abstract Introduction Precision medicine with minimal side effects remains the goal of cancer therapy. Iron oxide nanoparticles are non-toxic, successful carriers substances, targeting tissues and delivering high payloads conjugated agents. Interfering RNA (siRNA) disrupts protein expression, an effective treatment e.g. for hereditary amyloidosis. This study tested transferrin-conjugated dopami...