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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...
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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...
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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...
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Genetics and epigenetics: paternal adolescent ethanol consumption in serotonin transporter knock-out rats and offspring sensitivity to ethanol
RationaleAlcohol use disorder (AUD) is shown to have an overall heritability of around 50%. One of the genes associated with AUD is SLC6A4 (solute...
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Nanodelivery in Gene Therapy
Gene therapy stands as a revolutionary approach in medicine, aiming to rectify genetic abnormalities by introducing therapeutic genes or regulating... -
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...
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Emerging Model Organisms
This volume discusses different approaches towards understanding the brain plasticity, aging, and regeneration of the nervous system in model...
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Gene Editing Tool for Neurodegenerative Diseases
Neurodegenerative diseases (NDs) encompass a heterogeneous group of disorders characterized by the progressive degeneration and death of neural... -
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... -
Immune Gene Therapy of Cancer
Cancer is the second global cause of mortality. Conventional treatments, such as chemotherapy and radiotherapy, have not provided desirable... -
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... -
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...
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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... -
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...
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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... -
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...
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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...
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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 −/− ...
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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...
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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...