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Showing 1–7 of 7 results
Advanced filters: Author: Ali Nowrouzi Clear advanced filters

  • An adeno-associated virus (AAV) vector encoding a variant of human lipoprotein lipase was recently approved in Europe as the first gene therapy for the treatment of LPL deficiency. Here Manfred Schmidt and his colleagues report their analysis of AAV integration sites after injection of the gene therapy construct in LPL-deficient patients and in mice.

    • Christine Kaeppel
    • Stuart G Beattie
    • Manfred Schmidt
    Research
    Nature Medicine
    Volume: 19, P: 889-891

  • Immune system diversity is generated by V(D)J recombination, leading to clonal T-cell lineages. Here the authors investigate the events leading to T-cell diversity through the use of a modified PCR technique combined with deep sequencing.

    • Eliana Ruggiero
    • Jan P. Nicolay
    • Christof von Kalle
    ResearchOpen Access
    Nature Communications
    Volume: 6, P: 1-7

  • Zinc-finger nucleases allow targeted genetic modification at loci chosen by the investigator, but the extent of their off-target activity—which could be toxic to cells—has not been evaluated experimentally on a genome-wide scale. Gabriel et al. document the off- and on-target activity of zinc-finger nucleases using lentiviral vectors to tag integration sites.

    • Richard Gabriel
    • Angelo Lombardo
    • Christof von Kalle
    Research
    Nature Biotechnology
    Volume: 29, P: 816-823

  • In vivo reprogramming of somatic cells is hampered by the need for vectors to express the OKSM factors in selected organs. Here the authors report new AAV-based vectors capable of in vivo reprogramming at low doses.

    • Elena Senís
    • Lluc Mosteiro
    • Dirk Grimm
    ResearchOpen Access
    Nature Communications
    Volume: 9, P: 1-14

  • Adverse events stemming from the use of retroviral vectors in humans has prompted the search for methods predicting the fate and biological consequences of gene-modified cells after vector insertion. Methods of integration site analysis, such as linear amplification-mediated PCR (LAM-PCR), rely on use of restriction enzymes and identify only a fraction of all genomic integrants. This report describes a non–restriction enzyme–based LAM-PCR technique that provides comprehensive, unbiased integration site analysis.

    • Richard Gabriel
    • Ralph Eckenberg
    • Manfred Schmidt
    Research
    Nature Medicine
    Volume: 15, P: 1431-1436