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5 clusters · 13 sources · 18 days · First seen · Last updated

Gene-editing technology clinical progress and risks

Overview

Advancements in gene-editing technologies, including CRISPR-Cas9, base editing, and prime editing, are demonstrating significant therapeutic potential. Research from the Broad Institute indicates these tools are being utilized in over 25 clinical trials to target conditions such as leukemia, high cholesterol, and rare genetic diseases. Despite these clinical successes, research published in Nature by Columbia University highlights substantial risks regarding the use of these technologies in human embryos. While next-generation base editing has shown the ability to accurately modify DNA in single-cell embryos, the process also produces unpredictable changes. These biological risks currently prevent the clinical application of embryo editing. Recent developments have introduced a new technique termed ‘DNA pencil’ to address these safety concerns. Unlike traditional CRISPR-Cas9, which acts as molecular ‘scissors’ by cutting both strands of the DNA helix and risking genetic loss or mosaicism, this approach utilizes base editing. By attaching an enzyme to a modified Cas9 protein, researchers can convert a single chemical letter of the genome without breaking the double helix. In testing, this method was used to deactivate the NANOG gene and target the PCSK9 gene associated with heart disease. While these applications produced significantly fewer unintended genomic alterations than conventional methods, the technology still faces limitations, as some tested embryos exhibited chromosomal anomalies or unintended edits. In the commercial sector, CRISPR Therapeutics is working to evolve its capabilities beyond its approved medicine, Casgevy, by developing in-vivo editing processes. Additionally, its CTX460 program has entered phase 1 trials for alpha-1 antitrypsin deficiency. Adding to the debate on embryo modification, Origin Genomics CEO Cathy Tie has advocated for the development of technology to repair defective genes in human embryos. Using stem cells from donated embryos with life-threatening mutations, the startup aims to identify tools to prevent the inheritance of certain diseases.

Entities

Dieter Egli · He Jiankui · Vertex Pharmaceuticals · Stanford University · Columbia University Vagelos College of Physicians and Surgeons

Timeline

  1. [TECHNOLOGY] 2 sources
    Origin Genomics founder advocates for human embryo gene editing

    Origin Genomics founder Cathy Tie is advocating for the development of gene-edited human embryos to correct genetic diseases, despite significant regulatory bans and safety concerns regarding off-target effects

  2. [TECHNOLOGY] 5 sources
    New DNA pencil technique enables safer human embryo editing

    Researchers have tested a new “DNA pencil” base editing technique on human embryos, offering a safer, more precise alternative to traditional CRISPR-Cas9 by avoiding double-strand DNA breaks.

  3. [TECHNOLOGY] 2 sources
    Gene editing technology advances with new assembly methods and in-vivo targets

    New gene-editing developments, including the ‘prime assembly’ method for precise DNA insertion and CRISPR Therapeutics' move toward in-vivo treatments, are advancing the field of genomic medicine.

  4. [TECHNOLOGY] 2 sources
    Human embryo gene editing shows accuracy but carries clinical risks

    New research shows base editing can accurately modify human embryos, but unpredictable risks currently prevent clinical use, fueling ongoing ethical debates over heritable germline editing.

  5. [HEALTH] 2 sources
    Gene-editing technologies show clinical promise despite embryo research risks

    Gene-editing technologies like CRISPR and base editing show promise in clinical trials for cancer and rare diseases, but new research from Columbia University warns of significant risks in human embryo editing.

Sources

bandab.com.br · broadinstitute.org · celulastroncors.org.br · folhadoprogresso.com.br · informaparaiba.com.br · liveLaw.in · news-medical.net · nprinternedition.org · scientificinquirer.com · theascent.com · time.news · ultimosegundo.ig.com.br · wired.com

This summary has been updated 3 times: see revision history