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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human SCID-X1

2021-10-15

Abstract excerpt

<title>Abstract</title> <p>A 4–8 year follow-up of vector integration site (VIS) analysis in blood lineages from patients with X-linked severe combined immune deficiency receiving lentivector gene therapy found a > 60-fold increase in frequency of forward-orientated VIS within intron 3 of <italic>HMGA2</italic>. Some patients demonstrated emergence of dominant <italic>HMGA2</italic> VIS clones in progenitor and m...

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Literature Corpus work
2b45d822-6f21-5240-80ef-03c6f202a03b
DOI
10.21203/rs.3.rs-858393/v1
Open publication

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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human SCID-X1DOI 10.21203/rs.3.rs-858393/v1
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