Article

Development of a liver gene therapy strategy for haemophilia B with lentiviral vectors

2013-01-01

Abstract excerpt

Lentiviral vectors (LVs) are attractive tools for liver gene therapy, by virtue of their ability to stably integrate in the genome of target cells and the absence of pre-existing humoral and cellular immunity against vector components in most humans. We have previously reported long-term phenotypic correction of haemophilia B and transgene-specific immune tolerance induction after a single intravenous administrati...

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Literature Corpus work: 1e50362b-b520-52a6-aace-9f60b13356ed
DOI: 10.21954/ou.ro.0000f0bd

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