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Biotechnology: At the heart of gene edits in human embryos

Published on Aug 1, 2017in Nature 41.58
· DOI :10.1038/nature23533
Nerges Winblad1
Estimated H-index: 1
,
Fredrik Lanner20
Estimated H-index: 20
Abstract
The gene-editing technology CRISPR–Cas has been used in human embryos grown in vitro to correct a disease-associated mutation. The introduction of editing components at fertilization aided repair efficiency. See Article p.413
  • References (9)
  • Citations (3)
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References9
Newest
Published on Aug 1, 2017in Nature 41.58
Hong Ma27
Estimated H-index: 27
(Oregon National Primate Research Center),
Nuria Marti-Gutierrez1
Estimated H-index: 1
(Oregon Health & Science University)
+ 28 AuthorsRiffat Ahmed10
Estimated H-index: 10
(Oregon Health & Science University)
CRISPR–Cas9 genome editing is used to induce a DNA repair response and correct a disease-causing heterozygous mutation in human embryos with reduced mosaicism and preferential repair using the wild-type copy of the gene.
259 Citations Source Cite
Published on Jun 1, 2017in Molecular Genetics and Genomics 2.73
Lichun Tang1
Estimated H-index: 1
(Protein Sciences),
Yanting Zeng1
Estimated H-index: 1
(Guangzhou Medical University)
+ 8 AuthorsXiaowei Li1
Estimated H-index: 1
(Nanjing University)
Previous works using human tripronuclear zygotes suggested that the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system could be a tool in correcting disease-causing mutations. However, whether this system was applicable in normal human (dual pronuclear, 2PN) zygotes was unclear. Here we demonstrate that CRISPR/Cas9 is also effective as a gene-editing tool in human 2PN zygotes. By injection of Cas9 protein complexed with the appropriate sgRNAs and homology donors into o...
80 Citations Source Cite
Published on May 1, 2016in Journal of Assisted Reproduction and Genetics 2.79
Xiangjin Kang6
Estimated H-index: 6
(Guangzhou Medical University),
Wenyin He7
Estimated H-index: 7
(Guangzhou Medical University)
+ 5 AuthorsYong Fan12
Estimated H-index: 12
(Guangzhou Medical University)
Purpose As a powerful technology for genome engineering, the CRISPR/Cas system has been successfully applied to modify the genomes of various species. The purpose of this study was to evaluate the technology and establish principles for the introduction of precise genetic modifications in early human embryos.
118 Citations Source Cite
Published on Dec 1, 2015in Gene 2.50
Lucie Carrier43
Estimated H-index: 43
(University of Hamburg),
Giulia Mearini16
Estimated H-index: 16
(University of Hamburg)
+ 1 AuthorsFriederike Cuello6
Estimated H-index: 6
(University of Hamburg)
Abstract More than 350 individual MYPBC3 mutations have been identified in patients with inherited hypertrophic cardiomyopathy (HCM), thus representing 40–50% of all HCM mutations, making it the most frequently mutated gene in HCM. HCM is considered a disease of the sarcomere and is characterized by left ventricular hypertrophy, myocyte disarray and diastolic dysfunction. MYBPC3 encodes for the thick filament associated protein cardiac myosin-binding protein C (cMyBP-C), a signaling node in card...
46 Citations Source Cite
Published on May 1, 2015in Protein & Cell 6.23
Puping Liang7
Estimated H-index: 7
(Sun Yat-sen University),
Yanwen Xu14
Estimated H-index: 14
(Sun Yat-sen University)
+ 13 AuthorsYujing Li10
Estimated H-index: 10
(Sun Yat-sen University)
Genome editing tools such as the clustered regularly interspaced short palindromic repeat (CRISPR)-associated system (Cas) have been widely used to modify genes in model systems including animal zygotes and human cells, and hold tremendous promise for both basic research and clinical applications. To date, a serious knowledge gap remains in our understanding of DNA repair mechanisms in human early embryos, and in the efficiency and potential off-target effects of using technologies such as CRISP...
458 Citations Source Cite
Published on Dec 15, 2014in eLife 7.62
Steven Lin24
Estimated H-index: 24
(University of California, Berkeley),
Brett T. Staahl13
Estimated H-index: 13
(University of California, Berkeley)
+ 1 AuthorsJennifer A. Doudna92
Estimated H-index: 92
The CRISPR/Cas9 system is a robust genome editing technology that works in human cells, animals and plants based on the RNA-programmed DNA cleaving activity of the Cas9 enzyme. Building on previous work (Jinek et al., 2013), we show here that new genetic information can be introduced site-specifically and with high efficiency by homology-directed repair (HDR) of Cas9-induced site-specific double-strand DNA breaks using timed delivery of Cas9-guide RNA ribonucleoprotein (RNP) complexes. Cas9 RNP-...
397 Citations Source Cite
Published on Feb 15, 2013in Science 41.06
Le Cong15
Estimated H-index: 15
(Massachusetts Institute of Technology),
F. Ann Ran9
Estimated H-index: 9
(Massachusetts Institute of Technology)
+ 8 AuthorsLuciano A. Marraffini33
Estimated H-index: 33
(Rockefeller University)
Functional elucidation of causal genetic variants and elements requires precise genome editing technologies. The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage. We engineered two different type II CRISPR/Cas systems and demonstrate that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells. Cas9...
6,233 Citations Source Cite
Published on Aug 17, 2012in Science 41.06
Martin Jinek26
Estimated H-index: 26
(University of California, Berkeley),
Krzysztof Chylinski6
Estimated H-index: 6
(Umeå University)
+ 3 AuthorsEmmanuelle Charpentier28
Estimated H-index: 28
(Umeå University)
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems provide bacteria and archaea with adaptive immunity against viruses and plasmids by using CRISPR RNAs (crRNAs) to guide the silencing of invading nucleic acids. We show here that in a subset of these systems, the mature crRNA that is base-paired to trans-activating crRNA (tracrRNA) forms a two-RNA structure that directs the CRISPR-associated protein Cas9 to introduce double-stranded (ds) breaks in ...
4,942 Citations Source Cite
Published on Mar 13, 2002in JAMA 47.66
Barry J. Maron149
Estimated H-index: 149
ContextThroughout the past 40 years, a vast and sometimes contradictory literature has accumulated regarding hypertrophic cardiomyopathy (HCM), a genetic cardiac disease caused by a variety of mutations in genes encoding sarcomeric proteins and characterized by a broad and expanding clinical spectrum.ObjectivesTo clarify and summarize the relevant clinical issues and to profile rapidly evolving concepts regarding HCM.Data SourcesSystematic analysis of the relevant HCM literature, accessed throug...
1,756 Citations Source Cite
Cited By3
Newest
Liting You1
Estimated H-index: 1
(Sichuan University),
Ruizhan Tong2
Estimated H-index: 2
(Sichuan University)
+ 3 AuthorsYou Lu24
Estimated H-index: 24
(Sichuan University)
The expanding CRISPR-Cas9 technology is an easily accessible, programmable, and precise gene-editing tool with numerous applications, most notably in biomedical research. Together with advancements in genome and transcriptome sequencing in the era of metadata, genomic engineering with CRISPR-Cas9 meets the developmental requirements of precision medicine, and clinical tests using CRISPR-Cas9 are now possible. This review summarizes developments and established preclinical applications of CRISPR-...
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Published on May 1, 2019in Congenital Anomalies 1.15
Hideyo Ohuchi40
Estimated H-index: 40
(Okayama University),
Keita Sato5
Estimated H-index: 5
(Okayama University)
+ 2 AuthorsTetsuya Bando10
Estimated H-index: 10
(Okayama University)
2 Citations Source Cite
Published on Feb 1, 2019in American Biology Teacher 0.27
María José Apodaca2
Estimated H-index: 2
(National University of La Plata),
Joseph D. McInerney5
Estimated H-index: 5
(American Society of Human Genetics)
+ 2 AuthorsJorge V. Crisci21
Estimated H-index: 21
(National University of La Plata)
Is it possible to teach biology without mentioning evolution? The answer is yes, but it is not possible for students to understand biology without the evolutionary context on which the meaning and intellectual value of biological concepts depend. Meaningful learning of evolution requires (1) that the students incorporate new knowledge into a cognitive structure linked with higher-order concepts; (2) a well-organized knowledge structure; and (3) a positive emotional attachment and identification ...
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