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Nature, ISSN 0028-0836, 2013, Volume 500, Issue 7462, pp. 280 - 282
Focusing on three children with the disorder, Aiuti and colleagues used a similar approach to that of Biffiet al., but chose a different conditioning regimen.... 
Enzymes | Transplants & implants | Rodents | Cloning | Gene therapy | Cancer therapies | Patients | Blood | Cancer
Journal Article
by Schmidt, Manfred and Verhoeyen, Els and Gobbo, Emilie and Divers, Dominique and Oudrhiri, Noufissa and Griscelli, Frank and Bennaceur-Griscelli, Annelise and Klatzmann, David and Anguela, Xavier M and Sharma, Rajiv and Sharma, Rajiv and Li, Hojun and Li, Hojun and Haurigot, Virginia and Haurigot, Virginia and Haurigot, Virginia and Bhagwat, Anand and Bhagwat, Anand and Davidson, Robert and Davidson, Robert and Zhou, Shangzhen and Zhou, Shangzhen and Zhou, Shangzhen and Doyon, Yannick and Gregory, Philip D and Gregory, Philip D and Gregory, Philip D and Holmes, Michael C and Holmes, Michael C and Holmes, Michael C and High, Katherine A and Carbonaro, Denise and Shaw, Kit and Jin, Xiangyang and Geiger, Sabine and Mishra, Suparna and Cooper, Aaron and DeOliveira, Satiro and Sokolic, Rob and Candotti, Fabio and Carmo, Marlene and Arumugam, Paritha and Alonso-Ferrero, Maria and Schambach, Axel and Schambach, Axel and Baum, Christopher and Baum, Christopher and Baum, Christopher and Risma, Kimberly and Malik, Punam and Jordan, Michael and Rivat, Christine and Booth, Claire and Thrasher, Adrian and Whilding, Lynsey and Archibald, Kyra and Oberg, Daniel and Golan, Talia and Hubert, Ayala and Shemi, Amotz and Khvalevsky, Elina Zorde and Gabai-Malka, Racheli and Focht, Gili and Brunschwig, Zivia and Raskin, Stephen and Goldberg, Nahum and Ben-David, Eli and Peretz, Tamar and Eliakim, Rami and Dankur, Alan and Galun and Rachmur, Itzik and Domb, Avi and Kopelman, Yael and Hantz, Yael and Lahav, Mor and Arbel-Alon, Sagit and Dickson, George and Barkats, Martine and Daboussi, Fayza and Silva, Georges and Cedrone, Frederic and Epinat, Jean Charles and Juillerat, Alexandre and Valton, Julien and Montini, Eugenio and Biffi, Alessandra and Biffi, Alessandra and Calabria, Andrea and Calabria, Andrea and Biasco, Luca and Biasco, Luca and Cesani, Martina and Cesani, Martina and Benedicenti, Fabrizio and Benedicenti, Fabrizio and Plati, Tiziana and Leo, Simone and Zanetti, Gianluigi and Aiuti, Alessandro and ...
Human Gene Therapy, ISSN 1043-0342, 10/2012, Volume 23, Issue 10, pp. A1 - A173
Journal Article
Experimental Hematology, ISSN 0301-472X, 09/2017, Volume 53, pp. S35 - S35
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 03/2017, Volume 376, Issue 9, pp. 848 - 855
Journal Article
Human Gene Therapy, ISSN 1043-0342, 02/2016, Volume 27, Issue 2, pp. 148 - 165
Journal Article
Journal Article
Cancer, ISSN 0008-543X, 05/2017, Volume 123, Issue 10, pp. 1791 - 1799
Journal Article
Nature, ISSN 0028-0836, 09/2015, Volume 525, Issue 7569, pp. 380 - 383
Whether cancer is maintained by a small number of stem cells or is composed of proliferating cells with approximate phenotypic equivalency is a central... 
CANCER-CELLS | MAINTENANCE | ACTIVATION | HEMATOPOIESIS | PATHWAY | CITED2 | MULTIDISCIPLINARY SCIENCES | IMATINIB | STAT5 | DIFFERENTIATION | HIF-1-ALPHA | Piperazines - administration & dosage | Antineoplastic Combined Chemotherapy Protocols - administration & dosage | Cell Proliferation | Neoplastic Stem Cells - drug effects | Humans | Gene Expression Regulation, Neoplastic | Leukemia, Myelogenous, Chronic, BCR-ABL Positive - drug therapy | Leukemia, Myelogenous, Chronic, BCR-ABL Positive - genetics | PPAR gamma - metabolism | Thiazolidinediones - administration & dosage | Antineoplastic Combined Chemotherapy Protocols - pharmacology | STAT5 Transcription Factor - metabolism | Benzamides - administration & dosage | Thiazolidinediones - therapeutic use | Benzamides - therapeutic use | Basic Helix-Loop-Helix Transcription Factors - metabolism | Neoplastic Stem Cells - metabolism | Leukemia, Myelogenous, Chronic, BCR-ABL Positive - pathology | Neoplastic Stem Cells - pathology | Benzamides - pharmacology | Thiazolidinediones - pharmacology | Repressor Proteins - metabolism | Pyrimidines - administration & dosage | Piperazines - therapeutic use | Pyrimidines - pharmacology | Imatinib Mesylate | Piperazines - pharmacology | Antineoplastic Combined Chemotherapy Protocols - therapeutic use | Pyrimidines - therapeutic use | PPAR gamma - agonists | Leukemia, Myelogenous, Chronic, BCR-ABL Positive - metabolism | Trans-Activators - metabolism | Index Medicus | Life Sciences | Microbiology and Parasitology | Immunology | Bacteriology | Virology
Journal Article
Nature, ISSN 0028-0836, 09/2015, Volume 525, Issue 7569, p. 380
  Whether cancer is maintained by a small number of stem cells or is composed of proliferating cells with approximate phenotypic equivalency is a central... 
Cell receptors | Stem cells | Development and progression | Genetic aspects | Chronic myeloid leukemia | Health aspects | Agonists (Biochemistry) | Bone marrow | Cell division | Phosphorylation | Cytokines | Leukemia
Journal Article
Science, ISSN 0036-8075, 11/2009, Volume 326, Issue 5954, pp. 818 - 823
X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine... 
Myeloid cells | Genetic vectors | Demyelinating diseases | B lymphocytes | Stem cells | Hematocrit | Cell lines | Gene therapy | Cells | Hematopoietic stem cells | Research Article | HIV VECTORS | SEVERE COMBINED IMMUNODEFICIENCY | INTEGRATION SITE SELECTION | MICROGLIA | BONE-MARROW-TRANSPLANTATION | HUMAN CD34(+) CELLS | MULTIDISCIPLINARY SCIENCES | MOUSE MODEL | NOD/SCID MICE | LONG-TERM ENGRAFTMENT | DIFFERENTIATE | Myeloablative Agonists - therapeutic use | Genetic Therapy | Leukocytes, Mononuclear - metabolism | Microglia - metabolism | Humans | Adrenoleukodystrophy - pathology | Male | Transplantation, Autologous | ATP-Binding Cassette Transporters - genetics | Hematopoiesis | Adrenoleukodystrophy - therapy | Hematopoietic Stem Cells - physiology | Female | Cell Differentiation | Fatty Acids - blood | Hematopoietic Stem Cells - virology | Child | Microglia - cytology | Gene Expression | Transduction, Genetic | Hematopoietic Stem Cell Transplantation | ATP Binding Cassette Transporter, Sub-Family D, Member 1 | HIV-1 - genetics | Virus Integration | Disease Progression | Cell Lineage | Animals | Brain - pathology | Mice | Adrenoleukodystrophy - genetics | Transplantation Conditioning | Genetic Vectors | Usage | Care and treatment | Adrenoleukodystrophy | Patient outcomes | Transplantation | Health aspects | Viruses | Genetic disorders | Neurological disorders | ABCD1 protein | Index Medicus | Life Sciences | Cellular Biology
Journal Article
Thalassemia Reports, ISSN 2039-4357, 03/2013, Volume 3, Issue 1s, pp. 43 - e43
Oral presentation is available online 
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 04/2018, Volume 378, Issue 16, pp. 1479 - 1493
Gene therapy with CD34+ cells transduced with a lentivirus vector carrying a β-globin gene was performed in 22 patients. At a median of 26 months, all the... 
MEDICINE, GENERAL & INTERNAL | HEMOGLOBINOPATHIES | GLOBIN GENE | EFFICACY | BONE-MARROW-TRANSPLANTATION | ERYTHROPOIESIS | DISEASE | STEM-CELL TRANSPLANTATION | BURDEN | CONDITIONING REGIMEN | VECTOR | Index Medicus | Abridged Index Medicus
Journal Article
Experimental Physiology, ISSN 0958-0670, 07/2019, Volume 104, Issue 7, pp. 1074 - 1089
What is the central question of this study? Do Fog2 mice present a defect of small intestine homeostasis? What is the main finding and its importance? The... 
morphological adaptation | small intestine | FOG‐2 | retinoblastoma | crypt cell proliferation | Homeostasis | Physiological aspects | Analysis | Protein binding | Cell proliferation | Transcription factors | Adipose tissue | Retinoblastoma protein | Cell lineage | Villus | Insulin | Small intestine | Cofactors | High fat diet | Mutation | Retinoblastoma | Binding sites | Adipogenesis
Journal Article