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JAMA, ISSN 0098-7484, 04/2015, Volume 313, Issue 15, pp. 1550 - 1563
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 03/2017, Volume 376, Issue 9, pp. 848 - 855
Journal Article
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
Blood, ISSN 0006-4971, 08/2010, Volume 116, Issue 6, pp. 900 - 908
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Journal of Clinical Investigation, ISSN 0021-9738, 08/2007, Volume 117, Issue 8, pp. 2225 - 2232
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Human Gene Therapy, ISSN 1043-0342, 02/2016, Volume 27, Issue 2, pp. 148 - 165
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Journal of Clinical Investigation, ISSN 0021-9738, 09/2008, Volume 118, Issue 9, pp. 3132 - 3142
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 10/2014, Volume 371, Issue 15, pp. 1407 - 1417
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2016, Volume 138, Issue 3, pp. 920 - 924.e3
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The New England Journal of Medicine, ISSN 0028-4793, 04/2002, Volume 346, Issue 16, pp. 1185 - 1193
Journal Article