• Preclinical Evaluation of a Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV‐1 Infection

    9 days ago - By Wiley

    Preclinical evaluation of a novel transcription activator‐like effector nuclease to target the C‐C chemokine receptor type 5 locus. Up to 90% of CCR5 alleles are disrupted in primary CD4+ T cells without notable off‐target activity or impact on the potency of edited cells, paving their way for clinical application in hematopoietic stem and progenitor cells of HIV patients.
    Abstract
    Therapies to treat patients infected with human immunodeficiency virus aim at preventing viral replication but fail to eliminate the virus. Although transplantation of allogeneic CCR5Δ32 homozygous stem cell...
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