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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

BV-101 is an adeno- associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons and contributes to the clearance of the mutant huntingtin protein. In the UK, effort has been made to scale-up the country’s cell and gene therapy manufacturing capabilities.

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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.

DNA 52
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World RNA Day: What impact RNA it had on drug discovery?  

Drug Discovery World

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

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The use of base editing in stem-cell based therapies

Drug Discovery World

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Engineering antigen-specific primary human NK cells against HER-2 positive carcinomas.