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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. Lisa Yang and Hock E.

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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. By Sarah C.P. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

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The role of CRISPR in microbiome engineering breakthroughs

Drug Target Review

Using a synthetic guide RNA (gRNA), scientists can target a specific DNA sequence and employ Cas9 to cut it. Today, however, we will explore a different application of CRISPR: microbiome engineering… Clustered regularly interspaced short palindromic repeats (CRISPR) was first discovered in the genome of marine bacteria.

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Evolved prime editors are smaller and more efficient for therapeutic applications

Broad Institute

Now researchers at the Broad Institute of MIT and Harvard have used cutting-edge continuous laboratory evolution and engineering methods to develop improved versions of the gene-editing tool. Reverse transcriptase proteins that copy RNA templates into strands of DNA are found naturally in all plant and animal cells and in many viruses.

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Cas9 ancestor engineered into a compact genome editing tool

Broad Institute

Cas9 ancestor engineered into a compact genome editing tool By Tom Ulrich May 14, 2025 Breadcrumb Home Cas9 ancestor engineered into a compact genome editing tool Researchers adapt a compact RNA-guided enzyme from bacteria for a variety of DNA editing tasks in human cells.

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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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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor. This engineered capsid opens up a world of possibilities."

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