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Pushing the envelope with PCSK9

  1. Katie Kingwell
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PCSK9 inhibition is a well-validated lipid-lowering approach and, in addition, has become an important testing ground for various therapeutic modalities. Now, three studies add to this pioneering portfolio, demonstrating liver-selective PCSK9 targeting with CRISPR base editing in non-human primates, and with an oral antisense oligonucleotide (ASO) in animal models. Advances in these two platforms could have applications in a wide range of genetic conditions.

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Nature Reviews Drug Discovery 20, 506 (2021)

doi: https://doi.org/10.1038/d41573-021-00093-9

References

  1. Musunuru, K. et al. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates. Nature 593, 429–434 (2021)

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  2. Rothgangl, T. et al. In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels. Nat. Biotechnol. https://doi.org/10.1038/s41587-021-00933-4 (2021)

  3. Gennemark, P. et al. An oral antisense oligonucleotide for PCSK9 inhibition. Sci. Transl Med. 13, eabe9117 (2021)

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