BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld Science
  • BioWorld Asia
  • Data Snapshots
    • Biopharma
    • Medical technology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • NME Digest
  • Special reports
    • Infographics: Dynamic digital data analysis
    • BCI
    • Ebola outbreak
    • Hantavirus
    • Trump administration impacts
    • Med-tech outlook 2026
    • Under threat: mRNA vaccine research
    • BioWorld at 35
    • Biopharma M&A scorecard
    • Bioworld 2025 review
    • BioWorld MedTech 2025 review
    • BioWorld Science 2025 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
BioWorld - Monday, July 6, 2026
Breaking News: Science fiction realized: BCI tech is hereSee today's BioWorld Science
Home » Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA
To read the full story, subscribe or sign in.
Neurology/psychiatric

Efficient editing in skeletal muscle of dystrophic mice with SORT LNPs encapsulating Cas9 mRNA

March 25, 2026
No Comments
Gene editing holds promise for treating neuromuscular disorders such as limb-girdle muscular dystrophy, but its clinical translation remains challenging due to a lack of complementary delivery tools for the extensive network of skeletal muscles in the human body. A team at University of Massachusetts Chan Medical School compared editing outcomes mediated by either Cas9 mRNA and RNP delivery to skeletal muscle via local injection in the context of the previously described selective organ targeting (SORT) lipid nanoparticles (LNPs) platform.
BioWorld Science Genetic/congenital Neurology/psychiatric Gene therapy

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for July 2, 2026.
  • CINP 2026: Prenatal and adolescent stress shape brain vulnerability

    BioWorld Science
    Human biology is extraordinarily complex, and that sophistication emerges from the very beginning. During embryonic and fetal development, the organism’s...
  • AI-generated image for brain organoid neural research

    CINP 2026: organoids reveal autism and addiction mechanisms

    BioWorld Science
    At the 2026 World Congress of Neuropsychopharmacology (CINP), held in Glasgow June 26-29, 2026, researchers from Japan’s National Center of Neurology and...
  • Ontrack Therapeutics divulges compounds for neurodegenerative diseases

    BioWorld Science
    Ontrack Therapeutics Ltd. has reported new heterotricyclic compounds potentially useful for the treatment of neurodegenerative diseases.
  • Reunion Neuroscience reports new 5-HT2A partial agonists and/or 5-HT2B antagonists

    BioWorld Science
    Reunion Neuroscience Inc. has identified new 5-HT2A receptor partial agonists and/or 5-HT2B receptor antagonists potentially useful for the treatment of...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Medical technology
    • Newco news
    • Opinion
    • Regulatory
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/metabolic
    • Immune
    • Infection
    • Neurology/psychiatric
    • NME Digest
    • Patents
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2026. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing