During the last decade, an increased amount of oligonucleotides have been
developed as promising therapeutic agents for treatment of diseases. siRNA/shRNA is
one promising example of therapeutic oligonucleotides which can inhibit target gene
expression by degrading mRNA in a highly sequence-specific manner. Although,
siRNA shows potential therapeutic properties, the specific delivery remains a major
barrier for clinical development of siRNA-based drugs. In this chatper, we provide an
overview of current progress in the field of siRNA/shRNA delivery and discuss recent
patents and technical advances in the development of efficient small RNA delivery
vehicles including strategies to enhance their pharmacokinetic properties, to promote
their cellular uptake, and to foster corresponding clinical trials.
Keywords: Acute kidney injury, age-related macular edema, alpha v beta 3 integrin,
aptamer, aptamer siRNA chimera, C-C chemokine receptor type 5, cell-specific
ligands, chronic myeloid leukemia, clinical trials, familial adenomatous polyposis,
heregulin receptor, human immunodeficiency virus, hypercholersterolemia, nonarteric
ischemic optic neuropathy, non-targeted delivery, ocular hypertension
glaucoma, pachyonychia congenital, prostate specific membrane antigen, respiratory
syncytial virus, RNA interference, shRNA, siRNA, stable acid lipid particles,
transthyretin-mediated amyloidosis, viral siRNA delivery.
