Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

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De Ravin et al.report an unplanned interim analysis of a secondary safety outcome for an ongoing clinical trial on lentiviral gene therapy for the treatment of Body Sponge X-linked Severe Combined Immunodeficiency (NCT01306019).Vector induced alternative splicing events are identified that cause aberrant fusion transcripts, leading to clonal dominance Belt in a single patient and clonal expansion in others.This can be mitigated by the removal of the lentivector cryptic splice acceptor.