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Edited course of biomedical research: leaping forward with CRISPR

Published on Nov 1, 2017in Pharmacological Research 4.90
· DOI :10.1016/j.phrs.2017.09.008
Patrick J. Collins1
Estimated H-index: 1
(Amgen),
Christopher M. Hale13
Estimated H-index: 13
(Amgen),
Han Xu1
Estimated H-index: 1
(Amgen)
Abstract
Abstract Within the short few years since the report of its application in precise genome editing, CRISPR technology has become the method of choice to modify and modulate gene expression in biomedical research and therapeutic development. Subsequently, a variety of research, diagnostic, and therapeutic tools have been developed based upon CRISPR’s mechanism of action. Such tools have helped to deepen the understanding of fundamental biology and broaden the horizon in the search for treatments for diseases that have been considered hard or impossible to cure. As CRISPR technology advances closer to clinical applications, its short comings are becoming more apparent, thus creating opportunities to improve the technology’s efficacy, specificity, and safety profile in this setting. We will summarize the current status of CRISPR technology and discuss its future impact in this review.
  • References (85)
  • Citations (3)
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References85
Newest
Published on Jan 1, 2018in Journal of Cellular Biochemistry 2.96
Jamie L. Freiermuth1
Estimated H-index: 1
(Georgetown University),
Ian J. Powell-Castilla1
Estimated H-index: 1
(Georgetown University),
G. Ian Gallicano22
Estimated H-index: 22
(Georgetown University)
Human induced pluripotent stem cells (iPSCs) can be differentiated into any cell in the body unlocking enormous research potential. Combined with the recent discovery of CRISPR/Cas9 endonucleases in bacteria and their modification for use in biomedical research, these methods have the potential to revolutionize the field of genetic engineering and open the door to generating in vitro models that more closely resemble the in vivo system than ever before. Use of CRISPR/Cas9 has created a whirlwind...
4 Citations Source Cite
Published on Dec 1, 2017in Scientific Reports 4.12
Emmanouil Metzakopian9
Estimated H-index: 9
(Wellcome Trust Sanger Institute),
Alex Strong4
Estimated H-index: 4
(Wellcome Trust Sanger Institute)
+ 12 AuthorsJacob Lamberth1
Estimated H-index: 1
(Merck KGaA)
CRISPR-Cas9 technology has accelerated biological research becoming routine for many laboratories. It is rapidly replacing conventional gene editing techniques and has high utility for both genome-wide and gene-focussed applications. Here we present the first individually cloned CRISPR-Cas9 genome wide arrayed sgRNA libraries covering 17,166 human and 20,430 mouse genes at a complexity of 34,332 sgRNAs for human and 40,860 sgRNAs for the mouse genome. For flexibility in generating stable cell li...
10 Citations Source Cite
Published on Dec 1, 2017in Scientific Reports 4.12
V. Kapustianyk12
Estimated H-index: 12
,
Yu. Eliyashevskyy2
Estimated H-index: 2
+ 8 AuthorsRoman Gumeniuk10
Estimated H-index: 10
Hybrid organometallic systems offer a wide range of functionalities, including magnetoelectric (ME) interactions. However, the ability to design on-demand ME coupling remains challenging despite a variety of host-guest configurations and ME phases coexistence possibilities. Here, we report the effect of metal-ion substitution on the magnetic and electric properties in the paramagnetic ferroelectric NH2(CH3)2Al1−x Cr x (SO4)2 × 6H2O. Doing so we are able to induce and even tune a sign of the ME i...
28 Citations Source Cite
Published on Sep 22, 2017in Science 41.06
Dong Niu3
Estimated H-index: 3
(Zhejiang University),
Hong-Jiang Wei4
Estimated H-index: 4
(Yunnan Agricultural University)
+ 19 AuthorsEllen Shrock5
Estimated H-index: 5
(Harvard University)
Xenotransplantation is a promising strategy to alleviate the shortage of organs for human transplantation. In addition to the concerns about pig-to-human immunological compatibility, the risk of cross-species transmission of porcine endogenous retroviruses (PERVs) has impeded the clinical application of this approach. We previously demonstrated the feasibility of inactivating PERV activity in an immortalized pig cell line. We now confirm that PERVs infect human cells, and we observe the horizont...
128 Citations Source Cite
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 Jul 1, 2017in Science Advances
Jiyung Shin2
Estimated H-index: 2
(University of California, Berkeley),
Fuguo Jiang14
Estimated H-index: 14
(University of California, Berkeley)
+ 7 AuthorsJennifer A. Doudna92
Estimated H-index: 92
CRISPR (clustered regularly interspaced short palindromic repeats)–Cas9 gene editing technology is derived from a microbial adaptive immune system, where bacteriophages are often the intended target. Natural inhibitors of CRISPR-Cas9 enable phages to evade immunity and show promise in controlling Cas9-mediated gene editing in human cells. However, the mechanism of CRISPR-Cas9 inhibition is not known, and the potential applications for Cas9 inhibitor proteins in mammalian cells have not been full...
72 Citations Source Cite
Ana M. Moreno3
Estimated H-index: 3
(University of California, San Diego),
Prashant Mali29
Estimated H-index: 29
(University of California, San Diego)
Schematic of in vivo and ex vivo gene therapy modalities. In vivo gene therapy involves the direct intra‐tissue or systemic injection of delivery agents, followed by assaying of targeting efficacy with close monitoring of safety and efficacy of treatment. In this regard, re‐administration of delivery agents might be necessary to achieve therapeutic efficacy. A patient might also be treated via ex vivo gene therapy, where patient somatic cells are isolated and either (1) reprogrammed into patient...
11 Citations Source Cite
Published on Jul 1, 2017in Gene Therapy 3.20
Zaohua Huang3
Estimated H-index: 3
,
Asahi Tomitaka7
Estimated H-index: 7
+ 1 AuthorsMadhavan Nair39
Estimated H-index: 39
7 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 Jun 1, 2017in Nature Methods 26.92
Kellie A. Schaefer4
Estimated H-index: 4
,
Wen-Hsuan Wu7
Estimated H-index: 7
+ 3 AuthorsVinit B. Mahajan25
Estimated H-index: 25
132 Citations Source Cite
Cited By3
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Published on Mar 1, 2019in Leukemia 10.02
Olivier Humbert5
Estimated H-index: 5
(Fred Hutchinson Cancer Research Center),
George S. Laszlo11
Estimated H-index: 11
(Fred Hutchinson Cancer Research Center)
+ 7 AuthorsRoland B. Walter39
Estimated H-index: 39
(University of Washington)
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Published on Sep 1, 2018in Developmental Biology 3.26
S. Morgani3
Estimated H-index: 3
(Medical Research Council),
Nestor Saiz9
Estimated H-index: 9
(Memorial Sloan Kettering Cancer Center)
+ 7 AuthorsAnna-Katerina Hadjantonakis55
Estimated H-index: 55
(Cornell University)
Abstract The FGF/ERK signaling pathway is highly conserved throughout evolution and plays fundamental roles during embryonic development and in adult organisms. While a plethora of expression data exists for ligands, receptors and pathway regulators, we know little about the spatial organization or dynamics of signaling in individual cells within populations. To this end we developed a transcriptional readout of FGF/ERK activity by targeting a histone H2B-linked Venus fluorophore to the endogeno...
2 Citations Source Cite
Published on Jun 10, 2018
Wee Hong Woo1
Estimated H-index: 1
(Singapore Polytechnic)
As a biomedical science programme at polytechnic aims to provide a sound foundation in the biological sciences, the onus is on the curriculum developer to see that the relevance and currency of the programme content are justifiably challenging and work-ready. In other words, the programme needs to prepare its students adequately well for the intended industry. Since the inception of Human Genome Project, the molecular paradigm has been evolving. A biomedical science student who is oblivious to t...
1 Citations Source Cite