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Scientists develop RNA-targeting strategy to repair genetic cause of ALS and dementia

Medical Xpress

Scientists at University of Florida (UF) Scripps Biomedical Research have developed a potential medicine for a leading cause of ALS and dementia that works by eliminating disease-causing segments of RNA. The compound restored the health of neurons in the lab and rescued mice with the disease.

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Rare genetic disease caused by mutations in protein that controls RNA metabolism

Scienmag

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

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Targovax announces plans to rebrand as Circio

Pharmaceutical Technology

Targovax has unveiled plans to rebrand as Circio , reflecting its strategic shift to focus on expediting the development of its innovative circular RNA (circRNA) platform. Initially reported in 2011, CircRNA is a naturally occurring class of RNA.

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Scientists behind RNA-repairing strategy recognized with American Chemical Society’s Nobel Signature award

Scienmag

JUPITER, FL – The American Chemical Society announced that its prestigious 2022 Nobel Laureate Signature Award for Graduate Education in Chemistry honors a student from the Skaggs Graduate School of Chemical and Biological Sciences at Scripps Research and her faculty advisor, for their pioneering work developing RNA-targeting medications for genetic (..)

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General Design Methods for mRNA Drugs

Pharma Mirror

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. Scientists have been striving to optimize mRNA stability, immunogenicity, translation efficiency, and delivery systems to achieve efficient and safe mRNA delivery.

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Scientists implicate non-cardiac genes in congenital heart disease

Scienmag

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Then, organs develop and hopefully function properly.

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U-M RNA scientists identify many genes involved in neuron development

Scienmag

Credit: Nigel Michki Neurons result from a highly complex and unique series of cell divisions. For example, in fruit flies, the process starts with stem cells that divide into mother cells (progenitor cells), that then divide into precursor cells that eventually become neurons.

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