5,365 results found
    1. Cancer Biology
    2. Immunology and Inflammation

    Domain-swapped T cell receptors improve the safety of TCR gene therapy

    Michael T Bethune, Marvin H Gee ... David Baltimore
    Engineered T cell receptors enable targeted immunotherapy with a reduced risk of harmful autoimmunity.
    1. Cancer Biology
    2. Neuroscience

    Estrogen receptor alpha in the brain mediates tamoxifen-induced changes in physiology in mice

    Zhi Zhang, Jae Whan Park ... Stephanie M Correa
    Estrogen receptor alpha in the hypothalamus is required for the effects of chronic tamoxifen treament on gene expression, thermoregulation, bone, and movement in mice.
    1. Chromosomes and Gene Expression

    Therapeutic effects of telomerase in mice with pulmonary fibrosis induced by damage to the lungs and short telomeres

    Juan Manuel Povedano, Paula Martinez ... Maria A Blasco
    Telomerase gene therapy represents a novel effective treatment for pulmonary fibrosis associated with short telomeres by improving pulmonary function, decreasing inflammation and accelerating fiber disappearance in fibrotic lungs.
    1. Cell Biology
    2. Developmental Biology

    BRAF activates PAX3 to control muscle precursor cell migration during forelimb muscle development

    Jaeyoung Shin, Shuichi Watanabe ... Thomas Braun
    Genetic and biochemical approaches identify a new component of the cellular signaling machinery driving migration of limb muscle precursor cells during mouse embryogenesis and reveal the underlying molecular mechanism.
    1. Biochemistry and Chemical Biology
    2. Cancer Biology

    Multi-targeted therapy resistance via drug-induced secretome fucosylation

    Mark Borris D Aldonza, Junghwa Cha ... Yoosik Kim
    Targeted therapies induce an aberrant fucosylation of complex tumor secretomes stimulating the expansion of minority drug-resistant clones and promoting therapy resistance.
    1. Microbiology and Infectious Disease
    2. Structural Biology and Molecular Biophysics

    Structure of the gene therapy vector, adeno-associated virus with its cell receptor, AAVR

    Nancy L Meyer, Guiqing Hu ... Michael Stewart Chapman
    Cryo-electron microscopy reveals interactions at the interface between adeno-associated virus and its cellular receptor at 2.4Å resolution.
    1. Medicine
    2. Neuroscience

    AAV-Txnip prolongs cone survival and vision in mouse models of retinitis pigmentosa

    Yunlu Xue, Sean K Wang ... Constance L Cepko
    AAV-Txnip prolongs vision in mouse models of blindness, enhancing lactate catabolism, mitochondrial health, and ATP production in a condition that likely has a limited glucose supply.
    1. Neuroscience
    2. Stem Cells and Regenerative Medicine

    AAV ablates neurogenesis in the adult murine hippocampus

    Stephen Johnston, Sarah L Parylak ... Matthew Shtrahman
    Future work using recombinant AAV as an experimental tool in the dentate gyrus or as a gene therapy for diseases of the central nervous system should be carefully evaluated.
    1. Genetics and Genomics
    2. Neuroscience

    Rescue of behavioral and electrophysiological phenotypes in a Pitt-Hopkins syndrome mouse model by genetic restoration of Tcf4 expression

    Hyojin Kim, Eric B Gao ... Benjamin D Philpot
    Normalizing Tcf4 expression during early postnatal development improves behavioral outcomes in Pitt-Hopkins syndrome (PTHS) model mice, suggesting that genetic therapies are feasible in individuals with PTHS.
    1. Neuroscience

    Whole brain delivery of an instability-prone Mecp2 transgene improves behavioral and molecular pathological defects in mouse models of Rett syndrome

    Mirko Luoni, Serena Giannelli ... Vania Broccoli
    Global brain transduction of the instability-prone Mecp2 transgene by systemic AAV-PHP.eB administration is safe and effective in protecting male and female Mecp2 mutant mice from the Rett syndrome disease phenotype.

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