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The New England Journal of Medicine, ISSN 0028-4793, 03/2014, Volume 370, Issue 10, pp. 921 - 931
Journal Article
The New England Journal of Medicine, ISSN 0028-4793, 01/2009, Volume 360, Issue 5, pp. 447 - 458
Journal Article
Nature Genetics, ISSN 1061-4036, 11/2012, Volume 44, Issue 11, pp. 1243 - 1248
Journal Article
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 05/2017, Volume 127, Issue 5, pp. 1689 - 1699
BACKGROUND. Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and... 
MEDICINE, RESEARCH & EXPERIMENTAL | THERAPY | SCID PATIENTS | RETROVIRAL VECTORS | RECONSTITUTION | EXPRESSION | ADA | TRANSPLANTATION | Autografts | Genetic Therapy | Severe Combined Immunodeficiency - enzymology | Transduction, Genetic | Agammaglobulinemia - genetics | Adenosine Deaminase - genetics | Agammaglobulinemia - therapy | Humans | Adenosine Deaminase - biosynthesis | Child, Preschool | Hematopoietic Stem Cell Transplantation | Retroviridae | Severe Combined Immunodeficiency - therapy | Infant | Male | Gene Expression Regulation, Enzymologic | Severe Combined Immunodeficiency - genetics | Agammaglobulinemia - enzymology | Adolescent | Female | Adenosine Deaminase - deficiency | Genetic Vectors | Child | Treatment outcome | Care and treatment | Usage | Safety and security measures | Analysis | Adenosine deaminase deficiency | Genetic aspects | Genetic engineering | Transplantation | Research | Hematopoietic stem cells | Pediatrics | Intravenous administration | Severe combined immunodeficiency | Transplants & implants | ADA gene | Copy number | Stem cell transplantation | Clinical trials | Genomes | Immunology | Lymphocytes | Peripheral blood mononuclear cells | Genetics | Busulfan | Research centers | Age | CD34 antigen | Enzymes | Adenosine | Enrollments | Research & development--R&D | Immunodeficiency | Immune reconstitution | Data processing | Adenosine deaminase | Gene expression | Medical screening | Hemopoiesis | Children & youth | Medicine | Studies | Hospitals | Stem cells | Product development | Gene therapy | Leukocytes (granulocytic) | Mitogens | Index Medicus | Abridged Index Medicus
Journal Article
Nature, ISSN 0028-0836, 04/2017, Volume 544, Issue 7648, pp. 115 - 119
Transposable elements are viewed as 'selfish genetic elements', yet they contribute to gene regulation and genome evolution in diverse ways(1). More than half... 
CELLS | COMPLEX | REPETITIVE ELEMENTS | CIRCULAR RNAS | DOSAGE COMPENSATION | MULTIDISCIPLINARY SCIENCES | IN-VIVO | CROSS-LINKING | SEQUENCING DATA | IDENTIFICATION | WEB SERVER | RNA-Binding Proteins - genetics | Adenosine Deaminase - chemistry | Protein Biosynthesis | Adenosine Deaminase - metabolism | RNA Editing - genetics | Humans | Male | Neoplasm Proteins - metabolism | Protein Isoforms - isolation & purification | RNA-Binding Proteins - isolation & purification | RNA - genetics | Adenosine Deaminase - isolation & purification | Protein Isoforms - metabolism | Protein Isoforms - chemistry | DEAD-box RNA Helicases - deficiency | HEK293 Cells | Transcription, Genetic | RNA, Double-Stranded - metabolism | Adenosine Deaminase - deficiency | DEAD-box RNA Helicases - metabolism | Neoplasm Proteins - genetics | Nucleic Acid Conformation | Alu Elements - genetics | RNA, Double-Stranded - chemistry | RNA - metabolism | Inverted Repeat Sequences - genetics | Cell Line | Adenosine Deaminase - genetics | RNA-Binding Proteins - chemistry | Gene Expression Regulation | Mutagenesis - genetics | Exons - genetics | Genes, Reporter - genetics | RNA - chemistry | Genome, Human - genetics | DEAD-box RNA Helicases - genetics | RNA - biosynthesis | Animals | RNA, Double-Stranded - genetics | Protein Binding | Mice | Neoplasm Proteins - deficiency | RNA-Binding Proteins - metabolism | Evolution, Molecular | Protein Isoforms - genetics | Genetic research | Research | RNA | Human genome | Genomics | Methods | Proteins | Amino acids | Genomes | Stem cells | Index Medicus
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 06/2013, Volume 123, Issue 6, pp. 2703 - 2718
Journal Article