{
  "id": 159149,
  "title": "Virus-like particles deliver gene-editing tools into Duchenne mouse model",
  "url": "https://urgent.news/2026/08/05/virus-like-particles-deliver-gene-editing-tools-into-duchenne-mouse",
  "topic": "health",
  "section": "Health & Medicine",
  "published": "2026-08-05T04:00:06.000Z",
  "source": {
    "name": "The Transmitter",
    "slug": "the-transmitter",
    "url": "https://www.thetransmitter.org/spectrum/virus-like-particles-deliver-gene-editing-tools-into-duchenne-mouse-model/"
  },
  "original_language": "en",
  "account": "A new preprint study has revealed a promising method for delivering gene-editing tools to muscle cells in a mouse model of Duchenne muscular dystrophy (DMD). The researchers utilized virus-like particles (VLPs) that resemble viruses but lack genetic material, specifically a protein called dARC derived from fruit flies. These VLPs successfully delivered the Cas9 gene editor into muscle cells, enabling them to correct genetic errors and restore dystrophin expression.\n\nDuchenne muscular dystrophy is caused by variants in the DMD gene, resulting in the loss of dystrophin protein and progressive muscle weakness. In addition to muscle weakness, individuals with DMD are more likely to develop neurobehavioral conditions, including autism, which affects around 7 percent of the affected population.\n\nCurrent gene-delivery methods, such as lipid nanoparticles and adeno-associated viruses (AAVs), have limitations. AAVs, for instance, have size constraints and may trigger immune reactions, while lipid nanoparticles are difficult to direct towards organs beyond the liver. To overcome these obstacles, researchers are turning to VLPs that mimic viruses without their genetic content.\n\nThe study's lead investigator, Feng Zhang, professor of neuroscience at MIT, and his team focused on Drosophila ARC1 (dARC1), a gene that transfers mRNA across motor neuron-muscle fiber junctions. They engineered dARC proteins to self-assemble into virus-like shells when combined with mRNA, gRNA, and Cas9, creating a protective packaging that facilitated efficient gene editing.\n\nZhang and his colleagues tested the VLPs in E. coli and observed their ability to bind to muscle cells, as well as other tissues like the brain. The dARC VLPs demonstrated a strong affinity for SORCS2, a receptor present in multiple tissues. While the 18 percent dystrophin rescue observed in the mouse model is comparable to exon-skipping antisense oligonucleotides, the VLPs' instability in blood poses a challenge for systemic delivery.\n\nTo address this limitation, Zhang's lab is investigating ways to stabilize the particles in serum and explore similar VLPs for various therapeutic applications, including neurological diseases. The team aims to engineer the particles to engage with receptors that can cross the blood-brain barrier, potentially allowing for targeted delivery to the brain. An alternative approach would involve injecting the particles into cerebrospinal fluid, a strategy that could be optimized with further refinement of stability in biofluids.",
  "summary": "The approach uses a protein found in fruit flies to ferry the gene editor Cas9 into muscle cells.",
  "key_points": [
    "Virus-like particles (VLPs) deliver Cas9 gene editor to muscle cells in Duchenne mouse model.",
    "dARC protein from fruit flies assembles into VLPs for protective packaging.",
    "18% dystrophin rescue comparable to exon-skipping antisense oligonucleotides."
  ],
  "editors_take": null,
  "illustration": null,
  "coverage": {
    "outlets": 1,
    "also_reported_by": []
  },
  "ai_generated": true,
  "disclaimer": "Summaries, key points and the editor’s take are written by software from other outlets’ reporting and may contain errors — always check the linked original."
}