Sökning: "off-targets"

Hittade 4 avhandlingar innehållade ordet off-targets.

  1. 1. Targeted Long-read Sequencing : Development and Applications in Medical Genetics

    Författare :Ida Höijer; Ulf B. Gyllensten; Adam Ameur; Lars Feuk; Winston Timp; Uppsala universitet; []
    Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; long-read sequencing; targeted sequencing; targeted enrichment; CRISPR-Cas9; off-targets; medical genetics; repeat expansion; structural variant;

    Sammanfattning : Targeted sequencing has the advantage of providing pinpointed DNA information, while costs and data-analysis efforts are reduced. If targeted sequencing is combined with single molecule long-read sequencing, it can become a powerful tool to investigate genomic regions traditionally difficult using the predominantly used short-read sequencing platforms, including repetitive regions and large structural variants. LÄS MER

  3. 2. Improving integration efficiency and precision of CRISPR/Cas9-mediated genome editing

    Författare :Sandra Wimberger; Göteborgs universitet; []
    Nyckelord :NATURVETENSKAP; NATURAL SCIENCES; Genome editing; CRISPR-Cas9; DNA repair; genomic integration; off-targets;

    Sammanfattning : The clustered regularly interspaced short palindromic repeats (CRISPR) – CRISPR-associated protein 9 (Cas9) system has revolutionized the field of genome engineering, providing a cost-effective and fast tool for targeted gene modifications. Endogenous repair pathways, including error-prone non-homologous end joining and alternative end joining, or precise homology-directed repair (HDR), mend Cas9-induced DNA double-strand breaks. LÄS MER

  4. 3. Molecular Approaches to Explore Drug-Target Interactions

    Författare :Abdullah Al-Amin; Ulf Landegren; Christopher Schofield; Uppsala universitet; []
    Nyckelord :Drug discovery; target engagement; target engagement-mediated amplification; cellular thermal shift assay; proximity extension assay; in situ PLA; high-content imaging; Molekylär medicin; Molecular Medicine;

    Sammanfattning : Improved means to assess the clinical potential of drug candidates can critically influence development of new therapeutic entities, a central aim in medical life science. Drug discovery and development relies on construction and selection of small organic compounds or biological agents that bind targets of interest. LÄS MER

  5. 4. High resolution genomic tools for the discovery of protein function in mammalian cells

    Författare :Emily Carol Hodges; Karolinska Institutet; Karolinska Institutet; []
    Nyckelord :Cell-based assays; overexpression; RNAi; BC2; C3F; Taxol;

    Sammanfattning : The work described in this thesis emphasizes the development of cell-based strategies with increased throughput design that enable rapid biological discovery through direct investigation of protein function. Each of the works represented in this thesis are unified by the common premise that genetic perturbations, through loss or gain-of-function, in cell-based models may serve as an entry point for assigning biological roles to uncharacterized genes and their gene products. LÄS MER