• Knockout mice also offer a biological and scientific context in which drugs and other therapies can be developed and tested. (wikipedia.org)
  • They are widely used in knockout experiments, especially those investigating genetic questions that relate to human physiology. (wikipedia.org)
  • Consequently, observing the characteristics of knockout mice gives researchers information that can be used to better understand how a similar gene may cause or contribute to disease in humans. (wikipedia.org)
  • Examples of research in which knockout mice have been useful include studying and modeling different kinds of cancer, obesity, heart disease, diabetes, arthritis, substance abuse, anxiety, aging and Parkinson's disease. (wikipedia.org)
  • By causing a specific gene to be inactive in the mouse, and observing any differences from normal behaviour or physiology, researchers can infer its probable function. (wikipedia.org)
  • Many mouse models are named after the gene that has been inactivated. (wikipedia.org)
  • Other mouse models are named according to their physical characteristics or behaviours. (wikipedia.org)
  • It is time to harness new technologies and efficiencies of production to mount a high-throughput international effort to produce and phenotype knockouts for all mouse genes, and place these resources into the public domain. (nature.com)
  • To this end, highly immunodeficient mouse lines like the NOD.Cg- Prkdc scid Il2rg tm1Wjl /SzJ (NSG™, Stock# 005557 ) are an exciting and powerful development, particularly in understanding human immunology. (jax.org)
  • and (iii) the mouse genome supports targeted mutagenesis in specific genes by homologous recombination in embryonic stem (ES) cells, allowing genes to be altered efficiently and precisely. (nature.com)
  • The ultimate aim of the Knockout Mouse Project is to generate a null-mutant mouse resource comprising a null mutation marked with a reporter of high utility for each gene in mouse strain C57BL/6. (nih.gov)
  • To generate a novel CGD mouse model, Dr. Sweeney and colleagues used CRISPR/Cas9 methodology to create a 235 bp deletion in exon 1 of Cybb in the highly immunodeficient NSG mouse strain. (jax.org)
  • 2003). Previously, Cybb function was disrupted in C57BL/6 (B6.129S- Cybb tm1Din /J, stock# 002365 ) mice resulting in a mouse model of CGD (Pollock et al. (jax.org)
  • While NSG mice are highly deficient in lymphocytes, a limitation to using this strain is that NSG mice still maintain some phagocyte functionality, potentially confounding evaluation of therapies aimed at correcting defective phagocytes in engrafted human tissue. (jax.org)
  • The converging interests of multiple members of the genomics community led to a meeting to discuss the advisability and feasibility of a dedicated project to produce knockout alleles for all mouse genes and place them into the public domain. (nature.com)
  • The authors of this study demonstrate that fusing CRISPR/Cas9 technology with the highly immunodeficient NSG mouse is an exciting paradigm for the development of preclinical testing platforms specifically designed for greater translational relevance. (jax.org)
  • CRISPR-Mediated Knockout of Cybb in NSG Mice Establishes a Model of Chronic Granulomatous Disease for Human Stem-Cell Gene Therapy Transplants. (jax.org)
  • Now that the human and mouse genome sequences are known 1 , 2 , 3 , attention has turned to elucidating gene function and identifying gene products that might have therapeutic value. (nature.com)
  • Given that the vast majority of the human genome is transcribed, the mouse models developed by Sauvageau et al. (elifesciences.org)
  • this resulted in phagocytes that are incapable of ROS production in a mouse model that is capable of engrafting human hematopoietic cells. (jax.org)
  • As a proof of concept, CD34+ human hematopoietic stem cells from donors with or without CGD were engrafted in Cybb knockout mice. (jax.org)
  • When hematopoietic stem cells from CGD patients transduced with a lentiviral vector expressing functional human CYBB were engrafted into Cybb knockout mice, ROS activity was subsequently detected in phagocytes. (jax.org)
  • Mouse knockout technology provides a powerful means of elucidating gene function in vivo , and a publicly available genome-wide collection of mouse knockouts would be significantly enabling for biomedical discovery. (nature.com)
  • To investigate the functional relevance of lncRNAs in various physiological conditions, we have developed a collection of 18 lncRNA knockout strains in which the locus is maintained transcriptionally active. (elifesciences.org)
  • have developed several lines of knockout mice to investigate a subset of noncoding RNA molecules known as long intergenic noncoding RNAs (lincRNAs). (elifesciences.org)
  • Current technology also permits insertion of 'reporter' genes into the knocked-out gene, which can then be used to determine the temporal and spatial expression pattern of the knocked-out gene in mouse tissues. (nature.com)
  • Appreciation of the power of mouse genetics to inform the study of mammalian physiology and disease, coupled with the advent of the mouse genome sequence and the ease of producing mutated alleles, has catalyzed public and private sector initiatives to produce mouse mutants on a large scale, with the goal of eventually knocking out a substantial portion of the mouse genome 12 , 13 . (nature.com)
  • That these mice are capable of immune cell humanization enhances the translational relevance of the model. (jax.org)
  • The large-scale production and analysis of induced genetic mutations in worms, flies, zebrafish and mice have greatly accelerated the understanding of gene function in these organisms. (nature.com)
  • The curated Mouse Knockout & Mutation Database lists 2,669 unique genes (C. Rathbone, personal communication), the curated Mouse Genome Database lists 2,847 unique genes, and an analysis at Lexicon Genetics identified 2,492 unique genes (B.Z., unpublished data). (nature.com)
  • Analysis of peripheral blood phagocytes derived from Cybb knockout mice revealed that ROS production was abrogated. (jax.org)