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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. Previous research has shown that as little as 21 percent elimination of the protein can improve symptoms. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142
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Addressing increasingly resistant drugs by infectious agents

Drug Target Review

Infectious agents’ resistance was described at the European Union Research Parliament “as a widespread, multifaceted phenomenon that affects both living things and the environment, as well as their capacity to survive in the presence of drugs intended to either kill or inactivate them.”

Drugs 111
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Enhancing gene therapy with Circio

Drug Target Review

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

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The Future of Drug Discovery: Tackling the Undruggable with New Biotechnologies

DrugBank

This drug, which targets a specific protein-protein interaction (PPI), took over two decades of research and development but has proven highly effective in treating certain cancers. By blocking this interaction, researchers aim to unleash p53's full tumor-suppressing potential.

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The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

Conversations in Drug Development Trends

Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways. Recent Advancements in Biomarker Research The latest advancements in biomarker research have further elevated the biomarker’s role in disease diagnosis, treatment, and monitoring.

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Accelerating Riboswitch Engineering for the Development of Human Performance Biosensors - A Preview of Strateos’ Presentation at SynBioBeta 2021

The Strateos Blog: Drug Discovery

Learn how Strateos’ automated synthetic biology modules supported rapid, iterative screening of RNA sequences in modular pbuE expression system and E. coli extract-based cell-free protein expression assays.

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Marvin Caruthers receives inaugural Merkin Prize in ceremony at the Broad Institute for DNA synthesis technology

Broad Institute

Today, scientists use such reactions to produce the customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and more. Merkin praised Caruthers for the scope and impact of his prizewinning research.

DNA 52