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Optimized lentiviral vectors a solution for paediatric metabolic liver diseases

Fotios Sampaziotis1, Gloria Gonzalez-Aseguinolaza2

  • 1Cambridge Stem Cell Institute, Cambridge, UK; Cambridge Liver Unit, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK; National Institute of Health Research (NIHR) Blood and Transplant Research Unit (BTRU) at the University of Cambridge in Collaboration with Newcastle University and in Partnership with NHS Blood and Transplant (NHSBT), Cambridge, UK; Department of Medicine, University of Cambridge, Cambridge, UK; Centre for Translational Stem Cell Biology, Hong Kong, China.

Journal of Hepatology
|June 23, 2026
PubMed
Abstract

No abstract available in PubMed .

Keywords:
Isolated methylmalonic acidemia (MMA)Lentiviruspositive selective advantagesafe integration

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