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An ancient RNA-guided system could simplify delivery of gene editing therapies

Broad Institute

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

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A Visual Guide to Genome Editors

Codon

Certain CRISPR components can add short DNA sequences from the genomes of defeated viruses into the bacterium’s own genome, creating a type of protective “memory.” The core of the CRISPR immune response is a guide RNA (gRNA) that binds to a CRISPR-associated (Cas) protein. Visit our website to get a copy.

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Levers for Biological Progress

Codon

Scientists are already building a model that can, for example, look at which RNA molecules are expressed in a cell at t=0 and predict how those molecules will change at t=1. Synthesizing a single human protein-coding gene costs several hundred dollars and even a simple PCR machine (used for amplifying DNA) costs between $1,500 and $50,000.

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Twist Bioscience

Human Comprehensive Exome Human Core Exome Human Methylome Panel Human RefSeq Panel Mitochondrial Panel Mouse Exome Panel Respiratory Virus Research Panel Comprehensive Viral Research Panel Twist's Whole Exome Library Preparation Workflow.

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Using a Function-First “Scout Fragment”-Based Approach to Develop Allosteric Covalent Inhibitors of Conformationally Dynamic Helicase Mechanoenzymes

Covalent Modifiers

3c10581 Helicases, classified into six superfamilies, are mechanoenzymes that utilize energy derived from ATP hydrolysis to remodel DNA and RNA substrates. Kapoor Journal of the American Chemical Society 2024 DOI: 10.1021/jacs.3c10581 Therefore, helicases are important targets for chemical probes and therapeutics.

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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

Williams January 8, 2024 Credit: Susanna Hamilton, Broad Communications Researchers have developed virus-like particles that can deliver gene-editing cargo to cells, including those in the mouse brain. Prime editing, described in 2019 by Liu’s group, can make longer and more diverse types of DNA changes than other types of editing.

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Evolved prime editors are smaller and more efficient for therapeutic applications

Broad Institute

Their new editors are more efficient and specialized than previous versions, and are able to modify DNA in cultured cells and in animals that have been difficult to edit, including in immune system cells and inside the brain. What had not been done until now is improving the reverse transcriptase in a way that’s tailor-made for prime editing.”

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