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zadetkov: 84
1.
  • Use of adenine base editing... Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X
    Mention, Karen; Cavusoglu-Doran, Kader; Joynt, Anya T ... Human molecular genetics, 11/2023, Letnik: 32, Številka: 23
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    Abstract Small molecule drugs known as modulators can treat ~90% of people with cystic fibrosis (CF), but do not work for premature termination codon variants such as W1282X (c.3846G>A). Here we ...
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Dostopno za: UL
2.
  • Traditional stone water spo... Traditional stone water spouts status and its practical significance in urbanizing Kathmandu Valley, Nepal – a review
    Shrestha, Anustha; Shah, Deep Narayan; Bajracharya, Roshan Man ... Environmental challenges (Amsterdam, Netherlands), August 2022, 2022-08-00, 2022-08-01, Letnik: 8
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    •Traditional stone spouts can supplement the water demand in the Kathmandu valley if sufficiently conserved.•Rapid unplanned constructions and excessive groundwater extractions are affecting the ...
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3.
  • Practical Recommendations f... Practical Recommendations for Improving Efficiency and Accuracy of the CRISPR/Cas9 Genome Editing System
    Karagyaur, M. N.; Rubtsov, Y. P.; Vasiliev, P. A. ... Biochemistry (Moscow), 06/2018, Letnik: 83, Številka: 6
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    CRISPR/Cas9 genome-editing system is a powerful, fairly accurate, and efficient tool for modifying genomic DNA. Despite obvious advantages, it is not devoid of certain drawbacks, such as propensity ...
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Dostopno za: UL
4.
  • CRISPR/Cas9-mediated endoge... CRISPR/Cas9-mediated endogenous gene tagging in Fusarium oxysporum
    Wang, Qiang; Coleman, Jeffrey J. Fungal genetics and biology, 05/2019, Letnik: 126
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    Display omitted •Developed a CRISPR/Cas9-mediated EGT system for F. oxysporum.•First instance of a HITI strategy to insert a large fragment into the fungal genome.•FoChs5 showed polarized location at ...
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5.
  • Homology-independent target... Homology-independent targeted insertion (HITI) enables guided CAR knock-in and efficient clinical scale CAR-T cell manufacturing
    Balke-Want, Hyatt; Keerthi, Vimal; Gkitsas, Nikolaos ... Molecular cancer, 06/2023, Letnik: 22, Številka: 1
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    Chimeric Antigen Receptor (CAR) T cells are now standard of care (SOC) for some patients with B cell and plasma cell malignancies and could disrupt the therapeutic landscape of solid tumors. However, ...
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6.
  • Gene knock-out chain reacti... Gene knock-out chain reaction enables high disruption efficiency of HPV18 E6/E7 genes in cervical cancer cells
    Tian, Rui; Liu, Jiashuo; Fan, Weiwen ... Molecular therapy. Oncolytics, 03/2022, Letnik: 24
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    A genome editing tool targeting the high-risk human papillomavirus (HPV) oncogene is a promising therapeutic strategy to treat HPV-related cervical cancer. To improve gene knockout efficiency, we ...
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7.
  • Safe and effective liver-di... Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases
    Esposito, Federica; Dell’Aquila, Fabio; Rhiel, Manuel ... Cell reports. Medicine, 07/2024, Letnik: 5, Številka: 7
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    Liver-directed adeno-associated viral (AAV) vector-mediated homology-independent targeted integration (AAV-HITI) by CRISPR-Cas9 at the highly transcribed albumin locus is under investigation to ...
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8.
  • Genome editing in human hem... Genome editing in human hematopoietic stem and progenitor cells via CRISPR-Cas9-mediated homology-independent targeted integration
    Bloomer, Hanan; Smith, Richard H.; Hakami, Waleed ... Molecular therapy, 04/2021, Letnik: 29, Številka: 4
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    Ex vivo gene correction of hematopoietic stem and progenitor cells (HSPCs) has emerged as a promising therapeutic approach for treatment of inherited human blood disorders. Use of engineered ...
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9.
  • Long-term correction of hem... Long-term correction of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration
    Chen, Xi; Niu, Xuran; Liu, Yang ... Journal of genetics and genomics, December 2022, 2022-12-00, 20221201, Letnik: 49, Številka: 12
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    CRISPR/Cas9-mediated site-specific insertion of exogenous genes holds potential for clinical applications. However, it is still infeasible because homologous recombination (HR) is inefficient, ...
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10.
  • A comparison of DNA repair ... A comparison of DNA repair pathways to achieve a site-specific gene modification of the Bruton's tyrosine kinase gene
    Gray, David H.; Santos, Jasmine; Keir, Alexandra Grace ... Molecular therapy. Nucleic acids, 03/2022, Letnik: 27
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    Gene editing utilizing homology-directed repair has advanced significantly for many monogenic diseases of the hematopoietic system in recent years but has also been hindered by decreases between ...
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