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Gene expression delivery tool ‘slides’ instructions into cells

Drug Discovery World

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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Gene Accessibility May Explain Why Alzheimer’s Gene, APOE4, is Less Dangerous for People of African Descent

Drug Discovery Today

Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

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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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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.

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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Future Possibilities.

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Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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Moving the needle of CAR-T beyond oncology 

Drug Discovery World

DDW’s Megan Thomas speaks with Dr Blythe Sather, Vice President and Head of Research at Tune Therapeutics, about how genetic tuning will transform the reach for CAR-T therapies, the different diseases that can benefit, and the disruptive technology making this happen. MT: What is genetic tuning?