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Nucleic acids research, ISSN 1362-4962, 2009, Volume 37, Issue 6, pp. 1713 - 1725
Journal Article
Gene, ISSN 0378-1119, 08/2013, Volume 525, Issue 2, pp. 174 - 181
Substantial progress has been made in the past decade in treating several primary immunodeficiency disorders (PIDs) with gene therapy. Current approaches are... 
WAS | CGD | PID | Gene therapy | HSCT | SCID | REPERTOIRE | SEVERE COMBINED IMMUNODEFICIENCY | STEM-CELLS | CHRONIC GRANULOMATOUS-DISEASE | CELL TRANSPLANTATION | NEUTROPHILS | LENTIVIRAL VECTORS | TRANSGENE EXPRESSION | GENETICS & HEREDITY | INSERTIONAL MUTAGENESIS | RECONSTITUTION | Immunologic Deficiency Syndromes - therapy | Agammaglobulinemia - therapy | Humans | Hematopoietic Stem Cell Transplantation | Severe Combined Immunodeficiency - therapy | X-Linked Combined Immunodeficiency Diseases - therapy | Clinical Trials as Topic | Granulomatous Disease, Chronic - therapy | Transplantation, Autologous - methods | Hematopoietic Stem Cells - immunology | Immunologic Deficiency Syndromes - genetics | Mutagenesis, Insertional | Adenosine Deaminase - deficiency | Transgenes | Genetic Therapy - methods | Health aspects | Genes | Hematopoietic stem cells | Index Medicus | HLA, human leukocyte antigen | UCOE, ubiquitous chromatin opening element | PEG, polyethylene glycol | DSB, double strand break | SFFV, spleen focus-forming virus | EF-1α, elongation factor 1α | MDS, myelodysplastic syndrome | Review | PGK, phosphoglycerokinase | HSCs, haematopoietic stem cells | VSV-G, vesicular stomatitis virus-G protein | RVs, retroviral vectors | GALV, gibbon ape leukaemia virus | LCR, locus control region | TALEN, transcription activator-like effector nucleases | CGD, chronic granulomatous disorder | GvHD, graft versus host disease | GSH, genomic safe harbour | T-ALL, T cell-acute lymphoblastic leukaemia | ROS, reactive oxygen species | ADA-SCID, adenosine deaminase deficiency-severe combined immunodeficiency | NADPH, nicotinamide adenine dinucleotide phosphate hydrogen | LTR, long terminal repeat | IFN, interferon | LVs, lentiviral vectors | SIN, self-inactivating | miR, microRNA | XLN, X-linked neutropenia | HSCT, haematopoietic stem cell transplant | SAE, serious adverse event | WAS, Wiskott–Aldrich syndrome | HR, homologous recombination | SCID-X1, X-linked severe combined immunodeficiency | IL, interleukin | WASp, Wiskott–Aldrich syndrome protein | PIDs, primary immunodeficiency disorders | ERT, enzyme replacement therapy | MN, meganucleases | XLT, X-linked thrombocytopenia | MLV, murine leukaemia virus | NK cells, natural killer cells | ZFN, zinc finger nuclease
Journal Article
Catalysis Letters, ISSN 1011-372X, 6/2018, Volume 148, Issue 6, pp. 1723 - 1730
Journal Article
Seminars in immunology, ISSN 1044-5323, 2015, Volume 27, Issue 2, pp. 85 - 101
Highlights • Type I interferon represents the first line of innate host defense against a number of invading pathogens that also instructs adaptive immunity. •... 
Allergy and Immunology | Virus | Evasion mechanism | Signaling | Bacteria | Interferon | IFN-stimulated genes | Viruses - classification | Bacteria - immunology | Immune Evasion | Virus Diseases - immunology | Signal Transduction | Bacterial Infections - immunology | Bacteria - classification | Virus Diseases - virology | Viruses - immunology | Interferon Type I - immunology | Bacterial Infections - microbiology | WNV, West Nile virus | CPSF6, cleavage and polyadenylation specificity factor subunit 6 | IKK, inhibitor of kB kinase | SIV, simian immunodeficiency virus | CDNs, cyclic dinucleotides | JEV, Japanese encephalitis virus | AIM2, absent in melanoma 2 | pDCs, plasmacytoid dendritic cells | RT, reverse transcription | β, IFN-I | dsRNA, double-strand RNA | ITIM, immunoreceptor tyrosine-based inhibition motif | TIR, Toll-Interleukin-1 receptor | IFN-α | IFI16, interferon gamma-interferon-inducible protein 16 | MDA5, melanoma differentiation-associated gene-5 | ssDNA, single-stranded DNA | PLP, papain-like protease | TRAF, TNF receptor associated factor | MAPKs, mitogen-activated protein kinases | LRRFIP1, extrachromosomal histone | NF-κB, nuclear factor kappa-light-chain-enhancer of activated B cells | iNOS, inducible nitric oxide synthase 1 | dNTPase, deoxynucleotide triphosphate triphosphohydrolase | mRNA, messenger RNA | RNA, ribonucleic acid | miR, microRNA | TRIF, TIR domain-containing adaptor inducing IFN-β | SARS, severe acute respiratory syndrome | DAI, DNA-dependent activator of IFN-regulatory factors | TREX1, three prime repair exonuclease 1 | IFITM, IFN-induced transmembrane protein | STING, stimulator of IFN genes protein | TLRs, Toll-like receptors | NADPH, nicotinamide adenine dinucleotide phosphate | PACT, PKR-associated activator | MERS, Middle East respiratory syndrome | TRIM, tripartite motif | LPS, lipopolysaccharide | CypA, cyclophilin A | CoV, Coronavirus | IRF, IFN regulatory factor | ISRE, IFN-stimulated response element | PAMP, pathogen-associated molecular pattern | ISGF3, interferon-stimulated gene factor 3 | NSP, nonstructural protein | DCs, dendritic cells | MyD88, myeloid differentiation primary response gene 88 | CARD, caspase recruitment domain | HCV, hepatitis C virus | IRAK, IL-1R-associated kinases | DHX, DEAH box protein | CLRs, C-type lectin receptors | cIAP, cellular inhibitors of apoptosis | PKR, double-stranded RNA-activated protein kinase | RIP, receptor-interacting serine-threonine kinase | MAVS, mitochondrial antiviral signaling protein | ssRNA, single-stranded RNA | Type II IFN, IFN-II | DENV, dengue virus | H2B, leucine rich repeat (in FLII) interacting protein | NO, nitric oxide | SLFN11, schlafen family member 11 | SAMHD1, SAM domain and HD domain-containing protein 1 | DDX, DEAD box protein | tRNA, transfer RNA | DNA, deoxyribonucleic acid | ASC, apoptosis-associated speck-like protein containing a CARD | RIG-I, retinoic acid-inducible gene 1 | APOBEC, apolipoprotein B mRNA editing enzyme, catalytic polypeptide-like | IFN, interferon | PRR, pattern-recognition receptors | NLRP3, NACHT LRR and PYD domains-containing protein 3 | IFIT, IFN-induced tetratricopeptide repeat proteins | STAT, signal transducer and activator of transcription | cGAS, cyclic GMP-AMP synthase | NLRs, nucleotide-binding oligomerization domain-like receptors | RLRs, (RIG)-I-like receptors | HIV-1, human immunodeficiency virus 1 | NOD, nucleotide-binding oligomerization domain | MAM, mitochondrial associated membranes | SOCS, suppressor of cytokine signaling | JAK, Janus kinase | IPS1, IFN-β-promoter stimulator 1 | ISGs, IFN stimulated genes | ISG15, IFN-stimulated gene 15 | ER, endoplasmic reticulum | TBK1, TANK-binding kinase 1
Journal Article