Strategies for silencing human disease using RNA interference

DH Kim, JJ Rossi - Nature Reviews Genetics, 2007 - nature.com
Since the first description of RNA interference (RNAi) in animals less than a decade ago,
there has been rapid progress towards its use as a therapeutic modality against human …

Engineering siRNA therapeutics: challenges and strategies

SS Ali Zaidi, F Fatima, SA Ali Zaidi, D Zhou… - Journal of …, 2023 - Springer
Small interfering RNA (siRNA) is a potential method of gene silencing to target specific
genes. Although the US Food and Drug Administration (FDA) has approved multiple siRNA …

Gene delivery by lentivirus vectors

AS Cockrell, T Kafri - Molecular biotechnology, 2007 - Springer
The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …

Multifunctional cationic lipid-based nanoparticles facilitate endosomal escape and reduction-triggered cytosolic siRNA release

M Gujrati, A Malamas, T Shin, E **, Y Sun… - Molecular …, 2014 - ACS Publications
Small interfering RNA (siRNA) has garnered much attention in recent years as a promising
avenue for cancer gene therapy due to its ability to silence disease-related genes. Effective …

Emerging nanotechnology approaches for HIV/AIDS treatment and prevention

T Mamo, EA Moseman, N Kolishetti… - …, 2010 - Taylor & Francis
Currently, there is no cure and no preventive vaccine for HIV/AIDS. Combination
antiretroviral therapy has dramatically improved treatment, but it has to be taken for a …

HIV-1 TAR element is processed by Dicer to yield a viral micro-RNA involved in chromatin remodeling of the viral LTR

Z Klase, P Kale, R Winograd, MV Gupta… - BMC molecular …, 2007 - Springer
Background RNA interference (RNAi) is a regulatory mechanism conserved in higher
eukaryotes. The RNAi pathway generates small interfering RNA (siRNA) or micro RNA …

Gene therapy progress and prospects: RNA aptamers

NS Que-Gewirth, BA Sullenger - Gene therapy, 2007 - nature.com
Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high
affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug …

Exploring chemical modifications for siRNA therapeutics: a structural and functional outlook

S Shukla, CS Sumaria, PI Pradeepkumar - ChemMedChem, 2010 - Wiley Online Library
RNA interference (RNAi) is a post‐transcriptional gene silencing mechanism induced by
small interfering RNAs (siRNAs) and micro‐RNAs (miRNAs), and has proved to be one of …