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A deep dive into cells' RNA reality

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IMAGE: The researchers compared the BOLORAMIS method with single-molecule FISH (smFISH) analysis, a common standard for RNA localization studies, by tracing the location of a long non-coding RNA molecule known as.
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Credit: Wyss Institute at Harvard University
(BOSTON) ¬ Human cells typically transcribe half of their roughly 20,000 genes into RNA molecules at any given time. Just like with proteins, the function of those RNA species not only relies on their abundance but also their precise localization within the 3D space of each cell. Many RNA molecules convey gene information from the cell s nucleus to the protein-synthesizing machinery distributed throughout the cytoplasm (messenger RNAs or mRNAs), others are components of that machinery itself, while still different ones regulate genes and their expression, or have functions that remain to be discovered. Importantly, many diseases including cancer and neurological diseases have sign ....

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Novel computer-guided design tool helps increase the efficiency of cell conversions

Novel computer-guided design tool helps increase the efficiency of cell conversions
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A computational guide to lead cells down desired differentiation paths

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IMAGE: The collaborative team successfully used their computer-guided design tool IRENE to reconstruct the gene regulatory network controlling the identity of induced pluripotent stem cells (iPSCs).
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Credit: Wyss Institute at Harvard University
(BOSTON) There is a great need to generate various types of cells for use in new therapies to replace tissues that are lost due to disease or injuries, or for studies outside the human body to improve our understanding of how organs and tissues function in health and disease. Many of these efforts start with human induced pluripotent stem cells (iPSCs) that, in theory, have the capacity to differentiate into virtually any cell type in the right culture conditions. The 2012 Nobel Prize awarded to Shinya Yamanaka recognized his discovery of a strategy that can reprogram adult cells to become iPSCs by providing them with a defined set of gene-regulatory transcription factors (TFs). However, progres ....

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Machine-learning how to create better AAV gene delivery vehicles

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IMAGE: In their machine learning-based capsid diversification strategy, the team focused on a 28 amino acid peptide within a segment of the AAV2 VP3 capsid protein that exposes the AAV capsid.
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Credit: Wyss Institute at Harvard University (original by Drew Bryant)
(Boston) Adeno-associated viruses (AAVs) have become promising vehicles for delivering gene therapies to defective tissues in the human body because they are non-pathogenic and can transfer therapeutic DNA into target cells. However, while the first gene therapy products approved by the Federal Drug Administration (FDA) use AAV vectors and others are likely to follow, AAV vectors still have not reached their full potential to meet gene therapeutic challenges. ....

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