(Q106949811)

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

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

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1 reference

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1

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Alexandra C. Chadwick

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Sekar Kathiresan

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42

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author name string

Taiji Mizoguchi

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Sara P. Garcia

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Jamie E. DeNizio

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Caroline W. Reiss

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Kui Wang

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Sowmya Iyer

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Chaitali Dutta

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Victoria Clendaniel

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Michael Amaonye

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Aaron Beach

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Kathleen Berth

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Souvik Biswas

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Maurine C. Braun

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Huei-Mei Chen

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Thomas V. Colace

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John D. Ganey

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Soumyashree A. Gangopadhyay

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19

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Ryan Garrity

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Lisa N. Kasiewicz

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Jennifer Lavoie

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James A. Madsen

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Yuri Matsumoto

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Anne Marie Mazzola

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Yusuf S. Nasrullah

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Joseph Nneji

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Huilan Ren

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Athul Sanjeev

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Madeleine Shay

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Mary R. Stahley

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Steven H. Y. Fan

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Ying K. Tam

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33

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Nicole M. Gaudelli

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34

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Giuseppe Ciaramella

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35

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Leslie E. Stolz

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36

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Padma Malyala

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37

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Christopher J. Cheng

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38

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Kallanthottathil G. Rajeev

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39

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Ellen Rohde

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Andrew M. Bellinger

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41

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language of work or name

English

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publication date

19 May 2021

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full work available at URL

https://www.nature.com/articles/s41586-021-03534-y.pdf

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published in

Nature

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volume

593

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page(s)

429-434

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issue

7859

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describes a project that uses

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A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

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1

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Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system

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Engineering of CRISPR–Cas12b for human genome editing

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3

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Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

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4

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Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage.

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5

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The promise and challenge of therapeutic genome editing

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6

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Global, regional, and national age-sex-specific mortality for 282 causes of death in 195 countries and territories, 1980–2017: a systematic analysis for the Global Burden of Disease Study 2017

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7

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Search-and-replace genome editing without double-strand breaks or donor DNA

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8

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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

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9

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Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing

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Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing.

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11

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A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing.

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12

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In Vivo Base Editing of PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) as a Therapeutic Alternative to Genome Editing

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13

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Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

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14

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In utero CRISPR-mediated therapeutic editing of metabolic genes

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15

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Mutations in PCSK9 cause autosomal dominant hypercholesterolemia

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16

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Identifiers

DOI

10.1038/S41586-021-03534-Y

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ADS bibcode

2021Natur.593..429M

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(Q106949811)

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

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