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1. 1. Development of Gene Therapy for Hematopoietic Stem Cells using Lentiviral Vectors

   Författare :Niels-Bjarne Woods; Andreas Ooka; Stefan Karlsson; Avdelningen för molekylärmedicin och genterapi; []
   Nyckelord :MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; MEDICIN OCH HÄLSOVETENSKAP; MEDICAL AND HEALTH SCIENCES; embryonic stem cell; Hematopoietic stem cell; lentiviral vector; transduction efficiency; transgene expression; multiple vector copy integration; insertional mutagenesis; NOD SCID mouse; cytogenetics; Genetics; extracellulära vätskor; extracellular fluids; cytogenetik; Genetik; Haematology; Hematologi; Lentivirus : genetics; Human; Genetic Vectors; Hematopoietic Stem Cells : metabolism; Animal; Gene Therapy; Leukemia : therapy; Leukemia : metabolism;

   Sammanfattning : Hematopoietic stem cells are an ideal target for genetic manipulation for the purpose of curing hematological disorders as they have the ability to develop into all blood lineages and to self-renew. In this study we demonstrate that lentiviral vectors, based on HIV-1, can efficiently transfer genes into human hematopoietic progenitor and stem cells as assessed following stem cell transplantation in immune compromised mice. LÄS MER