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  1. Article

    Open Access

    GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy

    Lipid nanoparticles have demonstrated utility in hepatic delivery of a range of therapeutic modalities and typically deliver their cargo via low-density lipoprotein receptor-mediated endocytosis. For patients ...

    Lisa N. Kasiewicz, Souvik Biswas, Aaron Beach, Huilan Ren… in Nature Communications (2023)

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  2. No Access

    Article

    In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

    Gene-editing technologies, which include the CRISPR–Cas nucleases1–3 and CRISPR base editors4,5, have the potential to permanently modify disease-causing genes in patients6. The demonstration of durable editing i...

    Kiran Musunuru, Alexandra C. Chadwick, Taiji Mizoguchi, Sara P. Garcia… in Nature (2021)

  3. Article

    Open Access

    Development of an oral once-weekly drug delivery system for HIV antiretroviral therapy

    The efficacy of antiretroviral therapy is significantly compromised by medication non-adherence. Long-acting enteral systems that can ease the burden of daily adherence have not yet been developed. Here we des...

    Ameya R. Kirtane, Omar Abouzid, Daniel Minahan, Taylor Bensel… in Nature Communications (2018)

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  4. No Access

    Article

    A pH-responsive supramolecular polymer gel as an enteric elastomer for use in gastric devices

    Devices resident in the stomach—used for a variety of clinical applications including nutritional modulation for bariatrics, ingestible electronics for diagnosis and monitoring, and gastric-retentive dosage fo...

    Shiyi Zhang, Andrew M. Bellinger, Dean L. Glettig, Ross Barman… in Nature Materials (2015)

  5. No Access

    Article

    Hypernitrosylated ryanodine receptor calcium release channels are leaky in dystrophic muscle

    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 ch...

    Andrew M Bellinger, Steven Reiken, Christian Carlson, Marco Mongillo… in Nature Medicine (2009)