Abstract:
:Genome editing holds remarkable promise to transform human medicine as new therapies that can directly address the genetic causes of disease. However, concerns remain about possible undesired biological consequences of genome editors, particularly the introduction of unintended 'off-target' mutations. Here, we discuss both important considerations for therapeutic genome editing and our understanding of the functional impact of undesired off-target mutations. An important challenge for the future will be the development of new approaches for predicting and defining the probable function of unintended genome-editing mutations, which will inspire confidence in the next generation of promising genome-editing therapies.
journal_name
Genome Bioljournal_title
Genome biologyauthors
Cheng Y,Tsai SQdoi
10.1186/s13059-018-1610-2subject
Has Abstractpub_date
2018-12-22 00:00:00pages
226issue
1eissn
1474-7596issn
1474-760Xpii
10.1186/s13059-018-1610-2journal_volume
19pub_type
杂志文章相关文献
GENOME BIOLOGY文献大全abstract::As the cost of sequencing continues to decrease and the amount of sequence data generated grows, new paradigms for data storage and analysis are increasingly important. The relative scaling behavior of these evolving technologies will impact genomics research moving forward. ...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-016-0917-0
更新日期:2016-03-23 00:00:00
abstract:BACKGROUND:The human genome contains "dark" gene regions that cannot be adequately assembled or aligned using standard short-read sequencing technologies, preventing researchers from identifying mutations within these gene regions that may be relevant to human disease. Here, we identify regions with few mappable reads ...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-019-1707-2
更新日期:2019-05-20 00:00:00
abstract::The functional annotation of proteins identified in genome sequencing projects is based on similarities to homologs in the databases. As a result of the possible strategies for divergent evolution, homologous enzymes frequently do not catalyze the same reaction, and we conclude that assignment of function from sequenc...
journal_title:Genome biology
pub_type: 杂志文章,评审
doi:10.1186/gb-2000-1-5-reviews0005
更新日期:2000-01-01 00:00:00
abstract:BACKGROUND:There are significant limitations in existing methods for the genome-wide identification of genes whose expression patterns affect traits. RESULTS:The transcriptomes of five tissues from 27 genetically diverse maize inbred lines were deeply sequenced to identify genes exhibiting high and low levels of expre...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-017-1328-6
更新日期:2017-10-17 00:00:00
abstract:BACKGROUND:While the song of all songbirds is controlled by the same neural circuit, the hormone dependence of singing behavior varies greatly between species. For this reason, songbirds are ideal organisms to study ultimate and proximate mechanisms of hormone-dependent behavior and neuronal plasticity. RESULTS:We pre...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-014-0578-9
更新日期:2015-01-29 00:00:00
abstract::The spatial organization of chromatin in the nucleus has been implicated in regulating gene expression. Maps of high-frequency interactions between different segments of chromatin have revealed topologically associating domains (TADs), within which most of the regulatory interactions are thought to occur. TADs are not...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-019-1893-y
更新日期:2019-12-18 00:00:00
abstract::A report on the Wellcome Trust/Cold Spring Harbor Genome Informatics meeting, Cold Spring Harbor, USA, 7-11 May 2003. ...
journal_title:Genome biology
pub_type:
doi:10.1186/gb-2003-4-9-336
更新日期:2003-01-01 00:00:00
abstract::Big projects in biology - such as the human genome project and a number of related and ensuing enterprises - require big funding. A new tradition is growing in which some types of basic research take place within commercial organizations. This article reviews some of the reasons for this and some of the key players, i...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2002-3-4-comment2004
更新日期:2002-01-01 00:00:00
abstract:BACKGROUND:Transcription regulatory networks are composed of interactions between transcription factors and their target genes. Whereas unicellular networks have been studied extensively, metazoan transcription regulatory networks remain largely unexplored. Caenorhabditis elegans provides a powerful model to study such...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2005-6-13-r110
更新日期:2005-01-01 00:00:00
abstract:BACKGROUND:Four hypervariable minisatellite loci were scored on a panel of 116 individuals of various geographical origins representing a large part of the diversity present in house mouse subspecies. Internal structures of alleles were determined by minisatellite variant repeat mapping PCR to produce maps of interming...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2007-8-5-r80
更新日期:2007-01-01 00:00:00
abstract::The spectrum of mutations discovered in cancer genomes can be explained by the activity of a few elementary mutational processes. We present a novel probabilistic method, EMu, to infer the mutational signatures of these processes from a collection of sequenced tumors. EMu naturally incorporates the tumor-specific oppo...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2013-14-4-r39
更新日期:2013-04-29 00:00:00
abstract::Understanding the functional impact of genomic variants is a major goal of modern genetics and personalized medicine. Although many synonymous and non-coding variants act through altering the efficiency of pre-mRNA splicing, it is difficult to predict how these variants impact pre-mRNA splicing. Here, we describe a ma...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-018-1437-x
更新日期:2018-06-01 00:00:00
abstract::Map based cloning in Arabidopsis thaliana can be a difficult and time-consuming process, specifically if the phenotype is subtle and scoring labour intensive. Here, we have re-sequenced the 120-Mb genome of a novel Arabidopsis clock mutant early bird (ebi-1) in Wassilewskija (Ws-2). We demonstrate the utility of seque...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2011-12-3-r28
更新日期:2011-01-01 00:00:00
abstract:BACKGROUND:Gene duplications have a major role in the evolution of new biological functions. Theoretical studies often assume that a duplication per se is selectively neutral and that, following a duplication, one of the gene copies is freed from purifying (stabilizing) selection, which creates the potential for evolut...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2002-3-2-research0008
更新日期:2002-01-01 00:00:00
abstract:BACKGROUND:Malaria is a one of the most important infectious diseases and is caused by parasitic protozoa of the genus Plasmodium. Previously, quantitative characterization of the P. falciparum transcriptome demonstrated that the strictly controlled progression of these parasites through their intra-erythrocytic develo...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2008-9-12-r177
更新日期:2008-01-01 00:00:00
abstract:BACKGROUND:Previous methods employed for the identification of synergistic transcription factors (TFs) are based on either TF enrichment from co-regulated genes or phylogenetic footprinting. Despite the success of these methods, both have limitations. RESULTS:We propose a new strategy to identify synergistic TFs by fu...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2007-8-12-r257
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Obligate pathogenic bacteria lose more genes relative to facultative pathogens, which, in turn, lose more genes than free-living bacteria. It was suggested that the increased gene loss in obligate pathogens may be due to a reduction in the effectiveness of purifying selection. Less attention has been given t...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2007-8-8-r164
更新日期:2007-01-01 00:00:00
abstract:BACKGROUND:Differentiation of metazoan cells requires execution of different gene expression programs but recent single-cell transcriptome profiling has revealed considerable variation within cells of seeming identical phenotype. This brings into question the relationship between transcriptome states and cell phenotype...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-015-0683-4
更新日期:2015-06-09 00:00:00
abstract:BACKGROUND:The H subunit of the purple bacterial photosynthetic reaction center (PRC-H) is important for the assembly of the photosynthetic reaction center and appears to regulate electron transfer during the reduction of the secondary quinone. It contains a distinct cytoplasmic beta-barrel domain whose fold has no clo...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2002-3-11-research0061
更新日期:2002-10-14 00:00:00
abstract:BACKGROUND:DNA methylation and the Polycomb repression system are epigenetic mechanisms that play important roles in maintaining transcriptional repression. Recent evidence suggests that DNA methylation can attenuate the binding of Polycomb protein components to chromatin and thus plays a role in determining their geno...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2013-14-3-r25
更新日期:2013-03-25 00:00:00
abstract:BACKGROUND:Chromosome instability leads to aneuploidy, a state in which cells have abnormal numbers of chromosomes, and is found in two out of three cancers. In a chromosomal instable p53 deficient mouse model with accelerated lymphomagenesis, we previously observed whole chromosome copy number changes affecting all ly...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-016-0971-7
更新日期:2016-05-31 00:00:00
abstract::Many essential transcription factors have conserved roles in regulating biological programs, yet their genomic occupancy can diverge significantly. A new study demonstrates that such variations are primarily due to cis-regulatory sequences, rather than differences between the regulators or nuclear environments. ...
journal_title:Genome biology
pub_type: 杂志文章,评审
doi:10.1186/gb-2008-9-11-240
更新日期:2008-01-01 00:00:00
abstract:BACKGROUND:Synthetic genetic interactions have recently been mapped on a genome scale in the budding yeast Saccharomyces cerevisiae, providing a functional view of the central processes of eukaryotic life. Currently, comprehensive genetic interaction networks have not been determined for other species, and we therefore...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2012-13-7-r57
更新日期:2012-07-02 00:00:00
abstract::The circadian clock determines the rhythmic expression of many different genes throughout a 24-hour period. A recent study investigating the circadian regulation of liver proteins reveals multiple levels of regulation, including transcriptional, post-transcriptional and post-translational mechanisms. ...
journal_title:Genome biology
pub_type: 杂志文章,评审
doi:10.1186/gb-2006-7-9-234
更新日期:2006-01-01 00:00:00
abstract::Short hairpin RNAs can provide stable gene silencing via RNA interference. Recent studies have shown toxicity in vivo that appears to be related to saturation of the endogenous microRNA pathway. Will these findings limit the therapeutic use of such hairpins? ...
journal_title:Genome biology
pub_type: 杂志文章,评审
doi:10.1186/gb-2006-7-8-231
更新日期:2006-01-01 00:00:00
abstract::Two recent studies demonstrate the power of integrating tumor genotype information with epigenetic and proteomic studies to discover potential therapeutic targets in breast cancer. ...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-017-1238-7
更新日期:2017-05-22 00:00:00
abstract::Searching for similarities between biological sequences is the principal means by which bioinformatics contributes to our understanding of biology. Of the various informatics tools developed to accomplish this task, the most widely used is BLAST, the basic local alignment search tool. This article discusses the princi...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/gb-2001-2-10-reviews2002
更新日期:2001-01-01 00:00:00
abstract:BACKGROUND:Long noncoding enhancer RNAs (lnc-eRNAs) are a subset of stable eRNAs identified from annotated lncRNAs. They might act as enhancer activity-related therapeutic targets in cancer. However, the underlying mechanism of epigenetic activation and their function in cancer initiation and progression remain largely...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-020-02186-x
更新日期:2020-11-03 00:00:00
abstract::The first genome sequence of the extinct European wild aurochs reveals the genetic foundation of native British and Irish landraces of cattle.See related Research article: www.dx.doi.org/10.1186/s13059-015-0790-2. ...
journal_title:Genome biology
pub_type: 评论,杂志文章
doi:10.1186/s13059-015-0793-z
更新日期:2015-10-26 00:00:00
abstract::Studies of human genetic disorders have traditionally followed a reductionist paradigm. Traits are defined as Mendelian or complex based on family pedigree and population data, whereas alleles are deemed rare, common, benign, or deleterious based on their population frequencies. The availability of exome and genome da...
journal_title:Genome biology
pub_type: 杂志文章
doi:10.1186/s13059-016-1107-9
更新日期:2016-11-17 00:00:00