Innovative Technology that Enables RNAi in Difficult to Transfect Cells
Poster Apr 27, 2015
Christina Yamada, Kathryn Robinson, Allison St. Amand, Zaklina Strezoska, Greg Wardle, Anastasia Khvorova, Devin Leake
Delivery remains one of the last barriers for applying RNA interference (RNAi) in clinically relevant cell lines. Investigations at Dharmacon have led to the development of an innovative molecule for delivery in a wide variety of cell types. These modified siRNAs have been found to effectively silence target genes in cell types that are typically difficult to transfect using standard delivery methods. We present data for multiple cell types including SH-SY5Y (neuroblastoma), Jurkat (T-cells), and primary neurons. This technology, Dharmacon™ Accell™ siRNA reagents, allows for functional genomic studies in pertinent cell types. Moreover, in some instances, cells can be continuously dosed with these siRNAs, thus enabling knockdown of any target gene of interest for extended durations.
Inhibition of The Auto-inflammation Suppressor Protein ISG15 Triggers Preeclampsia by Blocking Trophoblast Migration and InvasionPoster
In summary, ISG15 expression levels are crucial for trophoblast morphology and function (migration/invasion). By blocking trophoblast invasion, reduced ISG15 levels could contribute to impaired spiral artery transformation that reduces utero-placental blood flow in preeclampsia. Thus, agents inducing ISG15 expression are likely to be therapeutic in preeclampsia.
An Emerging Phenotype of Partial RAG 1/2 Deficiency Among Young Children with Autoimmunity and Viral InfectionsPoster
We describe the natural history of a cohort of 12 patients with confirmed partial RAG1/2 mutations and autoimmunity at a young age. We were seeking the link between viral infections and autoimmunity and tested candidate biomarkers that may reflect the underlying RAG1/2 protein deficiency.READ MORE
Complete alignment identification of CRISPR-Cas9 genomic off-targets using Edit-R CRISPR specificity tool and a comprehensive analysis of positional mismatch tolerancePoster
A web tool that performs complete crRNA specificity checking is introduced. In addition, we evaluated positional off-targeting of the CRISPR system.READ MORE