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Three-in-one approach boosts the silencing power of CRISPR

Scienmag

A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. (..)

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A new strategy for siRNA stabilization by an artificial cationic oligosaccharide

Scienmag

Credit: TMIMS RNA interference is a gene regulatory mechanism in which the expression of specific genes is downregulated by endogenous microRNAs or by small interfering RNAs (siRNAs). Although siRNAs have broad potential for gene-silencing therapy, their instability is one of the difficulties to develop siRNA-based agents.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper.

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Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper.

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REGENERON AND ALNYLAM REPORT POSITIVE INTERIM PHASE 1 

The Pharma Data

These early results establish the first human translation of Alnylam’s proprietary C16-siRNA conjugate platform for central nervous system (CNS) delivery and are the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

It is important to highlight that IVT mRNAs are structurally similar to natural mRNAs and can be used to express proteins through genetic engineering. RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids.

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