Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- Author
- CARTIER, Nathalie1 2 ; HACEIN-BEY-ABINA, Salima3 4 5 ; MAHLAOUI, Nizar8 ; KIERMER, Véronique9 ; MITTELSTAEDT, Denice10 ; BELLESME, Céline2 ; LAHLOU, Najiba11 ; LEFRERE, François3 ; BIANCHE, Stéphane8 ; AUDIT, Muriel12 ; PAYEN, Emmanuel13 14 ; LEBOULCH, Philippe13 14 15 ; BARTHOLOMAE, Cynthia C6 ; L'HOMME, Bruno1 ; BOUGNERES, Pierre2 ; VON KALLE, Christof6 ; FISCHER, Alain4 8 ; CAVAZZANA-CALVO, Marina3 4 5 ; AUBOURG, Patrick1 2 ; VERSES, Gabor7 ; SCHMIDT, Manfred6 ; KUTSCHERA, Ina6 ; VIDAUD, Michel1 ; ABEL, Ulrich6 ; DAL-CORTIVO, Liliane3 5 ; CACCAVELLI, Laure3 5
[1] INSERM UMR745, University Paris-Descartes, 75279 Paris, France
[2] Department of Pediatric Endocrinology and Neurotogy, Hôpital Saint-Vincent de Paul, Assistance Publique-Hôpitaux de Paris, 82 Avenue Denfert-Rochereau, 75674 Paris, France
[3] Department of Biotherapy, Hôpital Necker-Enfants Malades, 75743 Paris, France
[4] INSERM UMR768, University Paris-Descartes, 75743 Paris, France
[5] Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris (Inserm), 75743 Paris, France
[6] National Center for Tumor Diseases and German Cancer Research Center (Deutsches Krebsforschungszentrum), 69120 Heidelberg, Germany
[7] Southwest 72nd Place, Gainesville, FL 32608, United States
[8] Department of Pediatric Immuno-Hematology, Hôpital Necker-Enfants Malades, 75743 Paris, France
[9] Nature Publishing Group, New York, NY 10013-1917, United States
[10] Quinton Road, San Diego, CA 92129, United States
[11] Department of Biochemistry, Hôpital Saint-Vincent de Paul, 75674 Paris, France
[12] Genosafe, 91002 Evry, France
[13] Commissariata L'Energie Atomique (CEA), Institute of Emerging Diseases and Innovative Therapies (iMETI), Fontenay-aux-Roses 92265, France
[14] INSERM U962 and Université de Paris XI, CEA-iMETI, Fontenay-aux-Roses 92265, France
[15] Genetics Division, Brigham and Women's Hospital and Harvard Medical School, Boston, MA 02115, United States - Source
Science (Washington, D.C.). 2009, Vol 326, Num 5954, pp 818-823, 6 p
- CODEN
- SCIEAS
- ISSN
- 0036-8075
- Scientific domain
- Multidisciplinary
- Publisher
- American Association for the Advancement of Science, Washington, DC
- Publication country
- United States
- Document type
- Article
- Language
- English
- Keyword (fr)
- Adrénoleucodystrophie Cellule hématopoïétique Cellule souche Greffe Homme Lentivirus Thérapie cellulaire Thérapie génique Vecteur Gène ABCD1 Endocrinopathie Enzymopathie Insuffisance surrénalienne Leucodystrophie Lipoïdose Maladie héréditaire Maladie métabolique Pathologie de l'encéphale Pathologie des surrénales Pathologie du système nerveux Retroviridae Sphingolipidose Virus
- Keyword (en)
- Adrenoleukodystrophy Hematopoietic cell Stem cell Graft Human Lentivirus Cell therapy Gene therapy Vector Endocrinopathy Enzymopathy Adrenal insufficiency Leukodystrophy Lipoidosis Genetic disease Metabolic diseases Cerebral disorder Adrenal gland diseases Nervous system diseases Retroviridae Sphingolipidosis Virus
- Keyword (es)
- Adrenoleucodistrofia Célula hematopoyética Célula primitiva Injerto Hombre Lentivirus Terapia celular Terapia génica Vector Endocrinopatía Enzimopatía Insuficiencia suprarrenal Leucodistrofia Lipoidosis Enfermedad hereditaria Metabolismo patología Encéfalo patología Suprarrenal patología Sistema nervioso patología Retroviridae Esfingolipidosis Virus
- Classification
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B21 Endocrinopathies / 002B21B Adrenals. Adrenal axis. Renin-angiotensin system (diseases) / 002B21B01 Non tumoral diseases. Target tissue resistance. Benign neoplasms
- Pascal
- 002 Biological and medical sciences / 002B Medical sciences / 002B22 Metabolic diseases / 002B22D Errors of metabolism / 002B22D02 Lipids (lysosomal enzyme disorders, storage diseases)
- Discipline
- Endocrinopathies Metabolic diseases
- Origin
- Inist-CNRS
- Database
- PASCAL
- INIST identifier
- 22119469
Sauf mention contraire ci-dessus, le contenu de cette notice bibliographique peut être utilisé dans le cadre d’une licence CC BY 4.0 Inist-CNRS / Unless otherwise stated above, the content of this bibliographic record may be used under a CC BY 4.0 licence by Inist-CNRS / A menos que se haya señalado antes, el contenido de este registro bibliográfico puede ser utilizado al amparo de una licencia CC BY 4.0 Inist-CNRS
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DOI
10.1126/science.1171242 
https://dx.doi.org/10.1126/science.1171242
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arXiv access
