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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
This review attempts to survey the potential to use traditional pharmaceuticals as opposed to biopharmaceuticals to treat single gene disorders. The available strategies include the use of antisense oligonucleotides to alter splicing or knock-down expression of a transcript, siRNAs to knock-down gene expression, and drugs for nonsense mutation read-through. 
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Neutralizing the RNA phosphodiester backbone enables delivery of siRNA across cell membranes.
BigField GEG Tech's insight:
The authors design the synthesis of short interfering ribonucleic neutrals (siRNNs) whose phosphate backbone contains neutral phosphotriester groups, allowing for delivery into cells. Once inside cells, siRNNs are converted by cytoplasmic thioesterases into native, charged phosphodiester-backbone siRNAs, which induce robust RNAi responses. geg-tech.com
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BigField GEG Tech's insight:
Here, the authors have used the CRISPR/Cas9 gene editing technology to create viable HEK293 derived cells lines that are deficient for a single catalytic subunit (STT3A or STT3B) or two STT3B-specific accessory subunits (MagT1 and TUSC3). Analysis of protein glycosylation in the STT3A, STT3B and MagT1/TUSC3 null cell lines revealed that these cell lines are superior tools for investigating the in vivo role and substrate preferences of the STT3A and STT3B complexes.
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BigField GEG Tech's insight:
CRISPR/Cas can be exploited not only to knockout genes but also to up- or down-regulate gene transcription—in some cases in a multiplex fashion. This provides a powerful tool for studying the interaction among multiple signaling cascades in the same genetic background.
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Differential knockdown of TGF-beta ligands in a three-dimensional co-culture ...
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Challenges in the delivery of small interfering RNA (siRNA) have hampered clinical translation. Polymeric or periodic short hairpin RNAs (p-shRNAs) are a recent development that can potentially address these delivery barriers by showing improved stability and complexation to enable nanoparticle packaging. Here The scientists modify these biomacromolecules packaging via structural and sequence engineering coupled with selective enzymatic digestion to generate an open-ended p-shRNA (op-shRNA) that is cleaved over ten times more efficiently to yield siRNA. The op-shRNA induces considerably greater gene silencing than p-shRNA in multiple cancer cell lines up to nine days. Op-shRNA provides an RNAi platform that can potentially be packaged and delivered efficiently to disease sites with higher therapeutic efficacy.