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NIH renews knockout mouse project for 3rd time

Drug Discovery World

Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. . Institutions in 15 countries.

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

Cell and gene therapies. There’s no question that cell and gene therapies have emerged as some of the most promising and innovative medicines on the market. . With 3,633 advanced therapeutical medicinal products (ATMPs) currently in some form of clinical or pre- clinical development, the sector shows no signs of slowing down.

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

Drug Discovery World

The NIH states: “This natural mechanism for sequence-specific gene silencing promises to revolutionise experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.” There are many examples of ways in which RNAi is impacting drug discovery.

RNA 52
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The use of base editing in stem-cell based therapies

Drug Discovery World

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage. 2016;353(6305):aaf8729. 2016;533, 420–424.