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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text

Medicine, Neurology, Neuroscience +17

Large-Scale Manufacture and Characterization of a Lentiviral Vector Produced for Clinical <i>Ex Vivo</i> Gene Therapy Application

Otto-Wilhelm Merten, Sabine Charrier, Nicolas Laroudie et al. · Human Gene Therapy · 2010 · 182 citations

Medicine, Biotechnology, Gene Therapy +13

Determination of Anti-Adeno-Associated Virus Vector Neutralizing Antibody Titer with an <i>In Vitro</i> Reporter System

Amine Meliani, Christian Leborgne, Sabrina Triffault et al. · Human Gene Therapy Methods · 2015 · 111 citations · Full text

Life Sciences, Biotechnology, Molecular Biology +12

Strategy to detect pre-existing immunity to AAV gene therapy

Lillian Falese, Krystal Sandza, Bridget Yates et al. · Gene Therapy · 2017 · 99 citations · Full text

Life Sciences, Hematology, Immunology +13

A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C

Serge Herson, Fayçal Hentati, Aude Rigolet et al. · Brain · 2011 · 87 citations

Medicine, Genetics, Molecular Medicine +14