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Novel approaches for correcting gene expression insufficiency

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Originally published by Olivia Dimmer, Northwestern University, on November 17, 2023   taRNAs built from an array of IRESs increase reporter gene translation. Credit: Nature Communications (2023). DOI: 10.1038/s41467-023-42252-z A new molecular technology capable of binding to mRNA and regulating gene expression may offer a new avenue for treating diseases caused by haploinsufficiency , or the absence of one functional gene copy, according to a study published in Nature Communications . Messenger RNA, or mRNA , contains instructions for DNA to produce proteins. Many diseases , including cancer and many genetic disorders, result from insufficient gene —and therefore protein—expression, but few strategies exist to correct that kind of dysregulation at a molecular level. The new technology, dubbed "translation-activating RNAs" ( taRNAs ), consists of small molecules programmed to attach to specific mRNA molecules to directly control their translation into proteins,...

A man's rare gene variant may have shielded him from devastating form of early Alzheimer's

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  Originally published by Stephanie Pappas on May 15, 2023 The discovery of a gene variant that protects against an early, insidious form of Alzheimer's could lead to new treatments for the disease.   A man carried a genetic mutation that set him up to develop early-onset Alzheimer's disease, but another gene kept him healthy for many years. (Image credit: byakkaya via Getty Images) A newly discovered genetic variant protects against a particularly devastating form of early Alzheimer's disease , raising scientists' hopes of finding treatments that can prevent or slow the progression of this and other forms of the disease.  Read more