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Gene expression delivery tool ‘slides’ instructions into cells

Drug Discovery World

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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Nucleome raises £37.5m to shine light on ‘dark genome’

pharmaphorum

million ($40 million) first-round financing that will be used to explore so-called ‘dark’ regions of the human genome. Nucleome’s platform adds 3D genomic information to a wealth of available genomic data, uncovering a new dimension of information that is disease as well as cell type-specific.

Genome 59
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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

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This week in drug discovery (2-6 October)  

Drug Discovery World

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52
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Epigenetics discovery could lead to new class of cancer drugs

Drug Discovery World

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

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Scientists uncover a new approach for treating aggressive cancer

The Pharma Data

UNC researchers now show that these two factors can directly associate with one another, modulating cancer-cell-specific programs of gene expression. ” They found that EZH2 possesses two different binding patterns on chromatin in acute leukemia cells, eliciting two distinct gene-regulatory programs (Figure 1).

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

Protein 52