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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.

RNA 66
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Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice

Broad Institute

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. 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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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases. we are first focused on engineering tRNAs to restore protein production in diseases caused by premature termination codons.

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The next phase of the multiomics evolution, powered by AI

Drug Target Review

The dynamic behaviour of RNA transcripts, the regulation and modification of proteins, the lipid composition of membranes, the structural organisation of cells and the spatial context of tissues all play critical roles in disease progression and therapeutic response.

RNA 71
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The role of CRISPR in microbiome engineering breakthroughs

Drug Target Review

When faced with a viral threat, bacterial cells developed an immune response by capturing and copying DNA fragments of viruses. This allowed bacteria to recognise subsequent attacks and cleave the viral DNA to stop the viral infection. It was also discovered that the Cas enzyme was responsible for DNA cleavage.

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Researchers uncover new CRISPR-like system in animals that can edit the human genome

Broad Institute

By Leah Eisenstadt June 28, 2023 Credit: Courtesy of the Zhang lab Cryo-EM map of a Fanzor protein in complex with ωRNA and its target DNA. They showed that Fanzor proteins use RNA as a guide to target DNA precisely, and that Fanzors can be reprogrammed to edit the genome of human cells. said Zhang.

RNA 139
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Building Korro Bio: A CEO’s Perspective on Innovation and Risk Management

LifeSciVC

Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing.

RNA 68