Strategies, design, and chemistry in siRNA delivery systems
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad
classes of diseases due to their ability to reversibly silence target genes. In August 2018, the …
classes of diseases due to their ability to reversibly silence target genes. In August 2018, the …
siRNA versus miRNA as therapeutics for gene silencing
Discovered a little over two decades ago, small interfering RNAs (siRNAs) and microRNAs
(miRNAs) are noncoding RNAs with important roles in gene regulation. They have recently …
(miRNAs) are noncoding RNAs with important roles in gene regulation. They have recently …
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones
Therapeutic small interfering RNA (siRNA) requires sugar and backbone modifications to
inhibit nuclease degradation. However, metabolic stabilization by phosphorothioate (PS) …
inhibit nuclease degradation. However, metabolic stabilization by phosphorothioate (PS) …
Current development of siRNA bioconjugates: from research to the clinic
IV Chernikov, VV Vlassov… - Frontiers in …, 2019 - frontiersin.org
Small interfering RNAs (siRNAs) acting via RNA interference mechanisms are able to
recognize a homologous mRNA sequence in the cell and induce its degradation. The main …
recognize a homologous mRNA sequence in the cell and induce its degradation. The main …
RNA interference (RNAi)-based therapeutics: delivering on the promise?
ML Bobbin, JJ Rossi - Annual review of pharmacology and …, 2016 - annualreviews.org
A resurgence in clinical trials using RNA interference (RNAi) occurred in 2012. Although
there were initial difficulties in achieving efficacious results with RNAi without toxic side …
there were initial difficulties in achieving efficacious results with RNAi without toxic side …
Nucleic acid degradation as barrier to gene delivery: a guide to understand and overcome nuclease activity
Gene therapy is on its way to revolutionize the treatment of both inherited and acquired
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …
From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization
MK Schlegel, MM Janas, Y Jiang, JD Barry… - Nucleic acids …, 2022 - academic.oup.com
Preclinical mechanistic studies have pointed towards RNA interference-mediated off-target
effects as a major driver of hepatotoxicity for GalNAc–siRNA conjugates. Here, we …
effects as a major driver of hepatotoxicity for GalNAc–siRNA conjugates. Here, we …
Action and reaction: the biological response to siRNA and its delivery vehicles
RNA interference (RNAi)-based therapeutics have significant potential for the treatment of
human disease. Safe and effective delivery of RNA to target tissues remains a major barrier …
human disease. Safe and effective delivery of RNA to target tissues remains a major barrier …
Editorial focus: understanding off-target effects as the key to successful RNAi therapy
R Bartoszewski, AF Sikorski - Cellular & molecular biology letters, 2019 - Springer
With the first RNA interference (RNAi) drug (ONPATTRO (patisiran)) on the market, we
witness the RNAi therapy field reaching a critical turning point, when further improvements in …
witness the RNAi therapy field reaching a critical turning point, when further improvements in …
Locked vs. unlocked nucleic acids (LNA vs. UNA): contrasting structures work towards common therapeutic goals
Oligonucleotide chemistry has been developed greatly over the past three decades, with
many advances in increasing nuclease resistance, enhancing duplex stability and assisting …
many advances in increasing nuclease resistance, enhancing duplex stability and assisting …