Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Florian Eichler(Harvard University), David A. Williams(Boston Children's Hospital), Jean‐Hugues Dalle(Inserm), Elizabeth McNeil(Inserm), Adrian J. Thrasher(Great Ormond Street Hospital), Himal L. Thakar(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Shaun A. Hussain(Inserm), Patricia L. Musolino(Harvard University), Jörn‐Sven Kühl(Charité - Universitätsmedizin Berlin), Satiro De Oliveira(University of California, Los Angeles), Troy C. Lund(University of Minnesota Medical Center), Juliana Folloni Fernandes(Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo), Nicholas Smith(Inserm), Paul J. Orchard(University of Minnesota), Caroline Sevin(Bicêtre Hospital), Laura A. Demopoulos(Inserm), Raman Sankar(Children’s Institute), Uwe Platzbecker(National Center for Tumor Diseases), Ashish O. Gupta(University of Minnesota), Paul Gissen(Great Ormond Street Hospital), Andrew C. Dietz(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Gerald Downey(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Christine Duncan(Dana-Farber/Boston Children's Cancer and Blood Disorders Center), Daniel J. Loes(Inserm), Hernán Amartino(Manchester Royal Eye Hospital), Patrick Aubourg(Inserm)
Cited by 71
Related Papers
Retinoic Acid and Arsenic Trioxide for Acute Promyelocytic Leukemia
|New England Journal of Medicine|2013|1.6k
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
|Science|2009|1.5k
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
|Nature|2010|1.3k
Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy
|New England Journal of Medicine|2002|1.1k
The cytoplasm of living cells behaves as a poroelastic material
|Nature Materials|2013|694