A Race Against Time: The Fight to Treat Duchenne Muscular Dystrophy

Published in Bioengineering & Biotechnology and Agricultural & Food Science

Go to the profile of ARGHYA SETT
ARGHYA SETT
Research Scientist, Luxembourg Inst. of Science and Technology
A Race Against Time: The Fight to Treat Duchenne Muscular Dystrophy
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Nature
Nature Nature

Aptamer-conjugated gold nanoparticles enable oligonucleotide delivery into muscle stem cells to promote regeneration of dystrophic muscles - Nature Communications

Lack of muscle stem cell targeting limits the treatment of muscular dystrophies. Here, the authors describe a platform based on functionalized gold nanoparticles that allows selective delivery of therapeutic oligonucleotides and promotes skeletal muscle regeneration.

Aptamer-conjugated gold nanoparticles offer a precise method for targeting muscle stem cells in Duchenne Muscular Dystrophy (DMD), addressing a major challenge in gene therapy. By ensuring microRNA therapies reach regenerative muscle cells with high specificity, this approach enhances treatment efficacy while minimizing off-target effects. Recent findings, published in Nature Communications, confirm that the platform is non-toxic and does not trigger immune reactions, marking a crucial step toward clinical translation and potential therapeutic applications.

https://www.nature.com/articles/s41467-024-55223-9

Go to the profile of ARGHYA SETT
ARGHYA SETT
Research Scientist, Luxembourg Inst. of Science and Technology

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Biomedical Engineering and Bioengineering
Technology and Engineering > Biological and Physical Engineering > Biomedical Engineering and Bioengineering
Agriculture
Life Sciences > Biological Sciences > Agriculture

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