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Antisense therapy restores fragile X protein production in human cells

Science Daily: Pharmacology News

This breakthrough was possible because of the novel findings, also presented in the study, that aberrant alternative splicing of messenger RNA (mRNA) plays a principal role in fragile X syndrome, the most common form of inherited intellectual disability and the most frequent single-gene cause of autism.

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RNA processing in health and disease: challenges and opportunities of the field

Drug Target Review

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

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Therapeutic Oligos 2025 Keynote Speakers Announced

Elrig

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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Researchers uncover new CRISPR-like system in animals that can edit the human genome

Broad Institute

They showed that Fanzor proteins use RNA as a guide to target DNA precisely, and that Fanzors can be reprogrammed to edit the genome of human cells. The new study demonstrates that RNA-guided DNA-cutting mechanisms are present across all kingdoms of life. said Zhang.

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Decoding stem cells for personalised regenerative medicine

Drug Target Review

Forging the toolkit for subcellular omics Cellular components known as organelles, consisting of RNA and protein, exert vital influences on human wellbeing and ailments by sustaining equilibrium, managing growth and ageing, and producing energy.

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Global Discovery of Covalent Modulators of Ribonucleoprotein Granules

Covalent Modifiers

Liganded sites were enriched for RNA-binding and protein–protein interaction (PPI) domains, including several sites found in RNP granule-forming proteins. In summary, we present a chemical strategy for the systematic discovery of condensate-modulating covalent small molecules.

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Role(s) of G3BPs in human pathogenesis [Minireview]

ASPET

Ras- G TPase-activating protein (SH 3 domain)- b inding p roteins (G3BP) are RNA binding proteins that plays a critical role in stress granule (SG) formation. Recent evidence suggests that G3BPs can also regulate mRNA expression through interactions with RNA outside of SGs.

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