Lentivector Cryptic Splicing Mediates Increase in CD34+ Clones Expressing Truncated HMGA2 in Human X-Linked Severe Combined Immunodeficiency

Researchers report vector insertion site (VIS) analysis in blood lineages from first eight treated patients with longer follow up found a > 60-fold increase in frequency of forward-orientated VIS within intron 3 of the High Mobility Group AT-hook 2 gene.
[Nature Communications]
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