Gene Therapy · 2011 · 59 citations · 45 references
PharmacyBiotechnologyCell BiologyCell TherapyGene DeliveryGene TherapyAnimal Cell CultureCell DeliveryCell EngraftmentGene Delivery To Stem CellsIn Vivo Gene DeliveryViral Vector-mediated TransgenesisCd34-positive Cell MobilizationLentiviral Gene TransferLentiviral IntegrationLentiviral Vector TransductionLentiviral VectorsRetroviral Vector TransductionTransduction Efficiency Optimization
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Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
Marina Cavazzana, Salima Hacein‐Bey, Geneviève de Saint Basile et al. · Science · 2000 · 2.6K citations
Life Sciences, Genetics, Immunology +17
NOD/SCID/γcnull mouse: an excellent recipient mouse model for engraftment of human cells
Mamoru Ito, Hidefumi Hiramatsu, Kimio Kobayashi et al. · Blood · 2002 · 1.5K citations
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana, Emmanuel Payen, Olivier Nègre et al. · Nature · 2010 · 1.3K citations