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Junk DNA: How the dark genome is changing RNA therapies

Drug Discovery World

Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. In actuality, the protein-coding portion of our genome is comparable in identity and number with the humble fruit fly or worm.

RNA 52
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Q&A: Gene therapy opportunities from long-read sequencing 

Drug Discovery World

Breakthroughs in gene therapy are only possible with an exact understanding of the genetic underpinnings of disease. To develop safe and effective gene therapies, researchers need confidence that genomic data is both complete and accurate.

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Why genomic healthcare data matters in the development of new therapies 

Drug Discovery World

One might argue that this all started getting exciting with the launch of the Human Genome Project, which the National Human Genome Research Institute consider to be one of the greatest scientific feats in history 1. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact. .

Genome 98
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Patients of African heritage have fewer actionable mutations

Drug Discovery World

“However, the extent to which differences in germline or somatic genomic alterations influence outcomes remains unknown.” Genetic sequencing To assess these alterations in groups of different ancestries, Walch and colleagues analysed targeted DNA sequencing data from 4,441 patients treated for colorectal cancer at MSK between 2014 and 2022.

Genome 52
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Researchers discover genetic cause of neurodevelopment disorders

Drug Discovery World

A global collaboration led by scientists at the University of Oxford has discovered that genetic variants in a specific gene cause a rare neurodevelopmental disorder (NDD). The specific gene is called RNU4-2. The study estimates that these specific changes in the RNU4-2 gene can explain 0.4%

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The pangenome is making personalised medicine more equitable

Pharmaceutical Technology

Basic human traits such as eye and hair colour are determined by our DNA. However, more immeasurable characteristics such as personality, behaviour, and even intelligence are all influenced by genetics to varying degrees. metres of supercoiled DNA contained within its nucleus. The first human genome cost $2.7bn to sequence.

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Genomic projects exploit scale as clinical applications play catch-up

Pharmaceutical Technology

The group analysed 12,222 samples collected through whole genome sequencing efforts of the UK National Health Service as part of the 100,000 Genomes Project and added further data on 6,418 cancers from the International Cancer Genome Consortium and the Hartwig Medical Foundation. Both teams had the same underlying goal.