In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

By Christopher E. Nelson, Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, E. Moreb, R. Rivera, S. Madhavan, Xiufang Pan, F. Ran, Winston X. Yan and 4 more. Science, 2016

1,069 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/268d6784fcd3fcf6ead2e1d353fa745fec9f3b0c

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Citations in the corpus

CRISPR-Cas9-based mutagenesis frequently provokes on-target mRNA misregulation (2019, positive): “These outcomes are currently achieved by using two CRISPR guides that flank a mutated exon 30-32 , or target an exon-specific splice junction using a single guide 33 .”
Identification of Pre-Existing Adaptive Immunity to Cas9 Proteins in Humans (2018, positive): “Much of the effort to treat disease in vivo has used Cas9 delivery systems that express the Cas9 protein for prolonged periods of time, such as through a non-integrating AAV vector or even through nanoparticle delivery of mRNA to non-dividing hepatocytes (8, 10-12).”
Cas-analyzer: an online tool for assessing genome editing results using NGS data (2016, methodology): “Contact: sangsubae@hanyang.ac.kr or jskim01@snu.ac.kr Supplementary information: Supplementary data are available at Bioinformatics online.”
Delivering CRISPR: a review of the challenges and approaches (2018, methodology): “Groups have used of SaCas9 in AAV vectors to target the cholesterol regulatory gene Pcsk9 (Ran et al., 2015) and disrupt mutations in the DMD gene in adult mice (Nelson et al., 2016; Tabebordbar et al., 2016).”
Engineering the Delivery System for CRISPR-based Genome Editing (2018, background): “While delivery of Cas9 RNPs results in the most immediate onset of gene editing, it can also be extremely transient, and suffers the additional challenge of delivery across two cellular barriers (the cell membrane and the nuclear membrane).”