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  1. Knock-out of 5-lipoxygenase in overexpressing tumor cells—consequences on gene expression and cellular function

    5-Lipoxygenase (5-LO), the central enzyme in the biosynthesis of leukotrienes, is frequently expressed in human solid malignancies even though the...

    Hannah Weisser, Tamara Göbel, ... Astrid S. Kahnt in Cancer Gene Therapy
    Article Open access 16 September 2022
  2. A tapt1 knock-out zebrafish line with aberrant lens development and impaired vision models human early-onset cataract

    Bi-allelic mutations in the gene coding for human trans-membrane anterior–posterior transformation protein 1 ( TAPT1 ) result in a broad phenotypic...

    Tamara Jarayseh, Brecht Guillemyn, ... Andy Willaert in Human Genetics
    Article 25 January 2023
  3. Protein O-GlcNAcylation and the regulation of energy homeostasis: lessons from knock-out mouse models

    O-GlcNAcylation corresponds to the addition of N-Acetylglucosamine (GlcNAc) on serine or threonine residues of cytosolic, nuclear and mitochondrial...

    Tarik Issad, Hasanain Al-Mukh, ... Patrick Pagesy in Journal of Biomedical Science
    Article Open access 04 September 2022
  4. Genetics and epigenetics: paternal adolescent ethanol consumption in serotonin transporter knock-out rats and offspring sensitivity to ethanol

    Rationale

    Alcohol use disorder (AUD) is shown to have an overall heritability of around 50%. One of the genes associated with AUD is SLC6A4 (solute...

    Sahir Hussain, Heidi M. D. Lesscher, ... Bart A. Ellenbroek in Psychopharmacology
    Article Open access 08 August 2022
  5. Nanodelivery in Gene Therapy

    Gene therapy stands as a revolutionary approach in medicine, aiming to rectify genetic abnormalities by introducing therapeutic genes or regulating...
    Orhan Burak Eksi, Aybuke Ulku Kutlu, ... Omer Aydin in Handbook of Cancer and Immunology
    Living reference work entry 2024
  6. Conditional knock out of transcription factor CTCF in excitatory neurons induces cognitive deficiency

    CCCTC-binding factor (CTCF) is a transcription factor that is involved in organizing chromatin structure. A reduction of CTCF expression is known to...

    Dong Il Choi, Myeongwon Kim, ... Bong-Kiun Kaang in Molecular Brain
    Article Open access 05 January 2021
  7. Emerging Model Organisms

    This volume discusses different approaches towards understanding the brain plasticity, aging, and regeneration of the nervous system in model...

    Wei Wang, Nicolas Rohner, Yongfu Wang in Neuromethods
    Book 2023
  8. Gene Editing Tool for Neurodegenerative Diseases

    Neurodegenerative diseases (NDs) encompass a heterogeneous group of disorders characterized by the progressive degeneration and death of neural...
    Mohd Yasir Khan, Hamda Khan, ... Arbab Husain in Mechanism and Genetic Susceptibility of Neurological Disorders
    Chapter 2024
  9. Virus-Induced Heritable Gene Editing in Plants

    Gene editing using clustered, regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) nuclease is an excellent tool for...
    Ugrappa Nagalakshmi, Nathan Meier, Savithramma P. Dinesh-Kumar in Plant-Virus Interactions
    Protocol 2024
  10. Immune Gene Therapy of Cancer

    Cancer is the second global cause of mortality. Conventional treatments, such as chemotherapy and radiotherapy, have not provided desirable...
    Abdolreza Esmaeilzadeh, Davood Jafari, ... Leila Karimi in Handbook of Cancer and Immunology
    Living reference work entry 2023
  11. Microfluidic Approaches for Gene Delivery and Therapy

    Gene editing techniques such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR/Cas9) have been developed to target a gene into...
    Mayur Parekh, Zulfiqur Ali in Microfluidics in Pharmaceutical Sciences
    Chapter 2024
  12. Circadian Clock Gene bmal1 Acts as a Tumor Suppressor Gene in a Mice Model of Human Glioblastoma

    Glioblastomas derived from malignant astrocytes are the most common primary tumors of the central nervous system in humans, exhibiting very bad...

    Laura L. Trebucq, Nicolas Salvatore, ... Juan J. Chiesa in Molecular Neurobiology
    Article 05 January 2024
  13. Genome Editing in CAR-T Cells Using CRISPR/Cas9 Technology

    CAR-T cell therapy is revolutionizing the treatment of hematologic malignancies. However, there are still many challenges ahead before CAR-T cells...
    Irene Andreu-Saumell, Alba Rodriguez-Garcia, Sonia Guedan in Cancer Immunotherapy
    Protocol 2024
  14. Targeted gene delivery systems for T-cell engineering

    T lymphocytes are indispensable for the host systems of defense against pathogens, tumors, and environmental threats. The therapeutic potential of...

    Fengling Wang, Yong Huang, ... Wei Wang in Cellular Oncology
    Article 16 May 2024
  15. Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases

    Prion disease is a diverse family of fatal and usually transmissible and progressive neurodegenerative diseases that strike humans and many other...
    Manuel Camacho, Qingzhong Kong in Prions and Diseases
    Chapter 2023
  16. Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight

    The remarkable advance in gene editing technology presents unparalleled opportunities for transforming medicine and finding cures for hereditary...

    Zhenjie Zhang, Siqi Zhang, ... Bo Feng in BioDrugs
    Article Open access 15 March 2024
  17. Successes and challenges in clinical gene therapy

    Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for...

    Donald B. Kohn, Yvonne Y. Chen, Melissa J. Spencer in Gene Therapy
    Article Open access 08 November 2023
  18. H2 influenza A virus is not pathogenic in Tmprss2 knock-out mice

    The host cell protease TMPRSS2 cleaves the influenza A virus (IAV) hemagglutinin (HA). Several reports have described resistance of Tmprss2 −/− ...

    Ruth Lydia Olga Lambertz, Ingo Gerhauser, ... Klaus Schughart in Virology Journal
    Article Open access 22 April 2020
  19. Gene editing technology to improve antitumor T-cell functions in adoptive immunotherapy

    Adoptive immunotherapy, in which tumor-reactive T cells are prepared in vitro for adoptive transfer to the patient, can induce an objective clinical...

    Yusuke Ito, Satoshi Inoue, Yuki Kagoya in Inflammation and Regeneration
    Article Open access 11 March 2024
  20. Ewing sarcoma treatment: a gene therapy approach

    Ewing sarcoma (ES) is an aggressive malignant tumor, characterized by non-random chromosomal translocations that produce fusion genes. Fusion genes...

    Sergey Tsibulnikov, Daria Fayzullina, ... Ilya Ulasov in Cancer Gene Therapy
    Article 10 April 2023
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