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Lipid nanoparticles and mRNA used to treat hereditary blindness

Drug Discovery World

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . It also doesn’t do fantastically well in continuing to express the enzymes the editing tool uses as molecular scissors to make cuts in the DNA to be edited.

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New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

The new technique controls gene activity without altering the DNA sequence of the genome by targeting chemical modifications that help package genes in our chromosomes and regulate their activity. The chemical modifications that regulate gene activity are called epigenetic markers.

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Why genomic healthcare data matters in the development of new therapies 

Drug Discovery World

From a general healthcare perspective, access to this sort of data is invaluable, but this is particularly noteworthy in that DNA tests have historically been limited in these regions on account of cost and availability of practitioners, services and resources.

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Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

Delveinsight

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

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Casting aside CRISPR scissors and making a point with base editors

pharmaphorum

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. Experts speak DDW asked a range of drug discovery experts where they thought the drug discovery expertise happening in the UK.

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