The discovery of the potential of small-interfering RNA (siRNA) has underscored the applications of RNA-based micro- and nanostructures in medicine.

Small-interfering selleck chem Dasatinib RNA (siRNA), synthetic double-stranded RNA consisting of approximately Inhibitors,Modulators,Libraries 21 base pairs, suppresses problematic target gem in a sequence-specific manner via inherent RNA interference (RNAi) processing As a result, siRNA offers a potential strategy for treatment of many human diseases. However, due to inefficient delivery to cells and off-target effects, the clinical application of therapeutic siRNA has been very challenging To address these issues, researchers have studied a variety of nanocarrier systems for siRNA delivery.

In this Account, we describe several strategies for efficient siRNA delivery and selective gene silencing.

Inhibitors,Modulators,Libraries We took advantage of facile chemical conjugation and complementary hybridization to design novel siRNA-based micro- and nanostructures. Using chemical crosslinkers and hydrophobic/hydrophilic polymers at the end of siRNA, we produced various RNA-based Inhibitors,Modulators,Libraries structures, including siRNA block copolymers, micelles, linear siRNA homopolymers, and microhydrogels. Because of their increased charge density and flexibility compared with conventional siRNA, these micro- and nanostructures can form polyelectrolyte complexes with poorly charged and biocompatible cationic carriers that are both more condensed and more homogenous than the complexes formed in other carrier systems. In addition, the fabricated siRNA-based structures are linked by cleavable disulfide bonds for facile generation of original siRNA in the cytosol and for target-specific gene silencing.

These newly developed siRNA-based structures greatly enhance intracellular uptake Inhibitors,Modulators,Libraries and gene silencing both in vitro and in vivo, making them promising biomaterials for siRNA therapeutics.”
“Twenty years after gene therapy was introduced in the clinic, advances in the technique continue to gamer headlines as successes pique the interest of clinicians, Batimastat researchers, and the public Gene therapy’s appeal stems from its potential to revolutionize modem medical therapeutics by offering solutions to myriad diseases through treatments tailored to a specific individual’s genetic code. Both viral and non-viral vectors have been used in the dinic, but the low transfection efficiencies when non-viral vectors are used have lead to an increased focus on engineering new gene delivery vectors.

To address the protein inhibitors challenges facing non-viral or synthetic vectors, specifically lipid-based carriers, we have focused on three main themes throughout our research: (1) The release of the nucleic acid from the carrier will increase gene transfection. (2) The use of biologically inspired designs, such as DNA binding proteins, to create lipids with peptide-based headgroups will improve delivery.