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Showing 1–5 of 5 results
Advanced filters: Author: Andrew M. Bellinger Clear advanced filters
  • Increased calcium levels in dystrophic muscle have damaging consequences. In this report, Bellinger et al. show that nitrosylation of the ryanodine receptor calcium channel, leading to calcium leak through the channel, is an underlying cause of increased calcium levels in the muscle of dystrophic mdx mice. Treatment of the mice with a compound that inhibits calcium leak increases their muscle function and physical activity, pointing to a potential new treatment for muscular dystrophy (pages 243–244).

    • Andrew M Bellinger
    • Steven Reiken
    • Andrew R Marks
    Research
    Nature Medicine
    Volume: 15, P: 325-330
  • In a cynomolgus macaque model, CRISPR base editors delivered in lipid nanoparticles are shown to efficiently and stably knock down PCSK9 in the liver to reduce levels of PCSK9 and low-density lipoprotein cholesterol in the blood.

    • Kiran Musunuru
    • Alexandra C. Chadwick
    • Sekar Kathiresan
    Research
    Nature
    Volume: 593, P: 429-434
  • Poor adherence to daily antiretrovirals can significantly affect treatment efficacy, but oral long-acting antiretrovirals are currently lacking. Here, the authors develop a once-weekly oral dosage form for anti-HIV drugs, assess its pharmacokinetics in pigs, and model its impact on viral resistance and disease epidemics.

    • Ameya R. Kirtane
    • Omar Abouzid
    • Giovanni Traverso
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-12