|1.||Chronic Granulomatous Disease
|2.||Wiskott-Aldrich Syndrome (Syndrome, Wiskott-Aldrich)
|3.||Adrenoleukodystrophy (Adrenoleukodystrophy, X-Linked)
|4.||Severe Combined Immunodeficiency (Bare Lymphocyte Syndrome)
|5.||T-Cell Leukemia (Leukemia, T Cell)
|1.||Thrasher, Adrian J: 9 articles (10/2014 - 04/2002)|
|2.||Felsburg, Peter J: 8 articles (03/2015 - 01/2002)|
|3.||Malech, Harry L: 8 articles (03/2015 - 07/2002)|
|4.||Leonard, Warren J: 8 articles (10/2011 - 11/2002)|
|5.||Hartnett, Brian J: 7 articles (07/2011 - 01/2002)|
|6.||Moore, Peter F: 6 articles (01/2014 - 04/2006)|
|7.||Henthorn, Paula S: 6 articles (07/2011 - 01/2002)|
|8.||De Ravin, Suk See: 5 articles (03/2015 - 07/2007)|
|9.||Kumaki, Satoru: 5 articles (09/2013 - 09/2002)|
|10.||Sorrentino, Brian P: 4 articles (03/2015 - 05/2009)|
|1.||Severe combined immunodeficiency due to adenosine deaminase deficiencyIBA
07/01/2003 - "Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. "
04/01/2014 - "Clinical results from the past 15 years have shown that significant improvement and even cure can be achieved for diseases such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease and Wiskott-Aldrich syndrome. "
05/01/2011 - "Retroviral vectors have been developed for hematopoietic stem cell (HSC) gene therapy and have successfully cured X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy, and Wiskott-Aldrich syndrome. "
01/01/2013 - "Retrovirus-based gene therapy was begun in 1990 for the patients of adenosine deaminase deficiency, followed by X-linked severe combined immunodeficiency, Wiskott-Aldrich syndrome and chronic granulomatous disease. "
12/01/2011 - "To date, HSC gene therapy has been successfully employed in the clinic for improving clinical outcomes in small numbers of patients with X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficiency (ADA-SCID), adrenoleukodystrophy (ALD), thalassemia, chronic granulomatous disease (CGD), and Wiskott-Aldrich syndrome (WAS). "
06/01/2012 - "Adenosine deaminase (ADA) deficiency, a cause of X-linked severe combined immunodeficiency (SCID), is a case in point. "
04/07/2003 - "Evidence of this progress is the recent successful functional correction of the immune T and B lymphocyte deficiency in patients with X-linked severe combined immunodeficiency (X-SCID) and adenosine deaminase (ADA)-deficient SCID following onco-retrovirus vector ex vivo transduction of autologous marrow stem cells [Science 296 (2002) 2410; Science 288 (2000) 669; N. "
04/01/2008 - "Results from clinical trials for X-linked severe combined immunodeficiency (SCID-X1), adenosine deaminase deficient SCID (ADA-SCID), and X-linked chronic granulomatous disease (X-CGD) are discussed. "
06/01/2009 - "Previous clinical trials based on the genetic correction of purified CD34(+) cells with gamma-retroviral vectors have demonstrated clinical efficacy in different monogenic diseases, including X-linked severe combined immunodeficiency, adenosine deaminase deficient severe combined immunodeficiency and chronic granulomatous disease. "
12/01/2014 - "Main focus is on the preclinical studies and clinical trials for the treatment of Wiskott-Aldrich syndrome, chronic granulomatous disease, adenosine deaminase deficient severe combined immunodeficiency (ADA-SCID) and X-linked severe combined immunodeficiency with lentiviral gene therapy. "
08/01/2012 - "Patients with X-linked severe combined immunodeficiency (SCID-X1) were successfully cured following gene therapy with a gamma-retroviral vector (gRV) expressing the common gamma chain of the interleukin-2 receptor (IL2RG). "
01/01/2015 - "We investigated the feasibility of interleukin-2 receptor gamma (IL2Rγ) gene based on gene mutation analysis and pre-natal diagnosis of X-linked severe combined immunodeficiency (X-SCID). "
08/01/2008 - "Here, we addressed the leukemogenic potential of vectors expressing interleukin-2 receptor common gamma-chain (IL2RG), the coding sequence required for correction of X-linked severe combined immunodeficiency. "
09/01/2007 - "Furthermore, an A2UCOE-IL2RG vector fully restored the IL-2 signaling pathway within IL2RG-deficient human cells in vitro and successfully rescued the X-linked severe combined immunodeficiency (SCID-X1) phenotype in a mouse model of this disease. "
12/01/1993 - "The molecular defect in X-linked severe combined immunodeficiency disease has been assigned to the gene encoding the gamma chain of the interleukin-2 receptor (IL-2R gamma), which is constitutively expressed by T cells and is involved in the formation of high and intermediate affinity IL-2R complexes. "
|5.||X-linked Severe combined immunodeficiencyIBA
02/01/2015 - "Samples were analyzed from transplanted pigtail macaques and from a participant in our X-linked severe combined immunodeficiency (XSCID) lentiviral vector trial and yielded controlled and quantitative results in all cases. "
09/01/1994 - "We describe a family affected by X linked severe combined immunodeficiency (SCIDX1) in which genetic prediction of carrier status was made using X chromosome inactivation studies together with limited genetic linkage analysis. "
04/01/1993 - "This study describes the thymic morphology in 52 dogs (ranging in age from 1 to 79 days) with X-linked severe combined immunodeficiency disease (XSCID). "
07/15/2011 - "We have previously shown that in vivo γ-retroviral gene therapy of dogs with X-linked severe combined immunodeficiency (XSCID) results in sustained T cell reconstitution and sustained marking in myeloid and B cells for up to 4 years with no evidence of any serious adverse effects. "
09/01/2008 - "X-linked severe combined immunodeficiency (XSCID) is caused by mutations of the common gamma chain (gammac) and usually characterized by the absence of T and natural killer (NK) cells. "
|6.||Complementary DNA (cDNA)IBA
06/12/2014 - "We demonstrate the therapeutic potential of our strategy by targeting a corrective complementary DNA into the IL2RG gene of HSCs from healthy donors and a subject with X-linked severe combined immunodeficiency (SCID-X1). "
12/01/2004 - "Eight of 10 infants with X-linked severe combined immunodeficiency who received autologous hematopoietic stem cells transduced with a retroviral vector carrying the IL2RG complementary DNA achieved immune reconstitution. "
02/01/2004 - "In this study, an MND-IL-2R vector containing IL-2Rc gamma cDNA to treat X-linked severe combined immunodeficiency (X-SCID) was constructed from an MND vector that was modified by substituting the myeloproliferative sarcoma virus (MPSV) enhancer for that of MoMLV, deleting the negative control region located in the long terminal repeat (LTR) as an enhancer, and replacing the primer binding site (PBS) of MoMLV with the PBS of the endogenous murine retrovirus dl587rev. "
10/09/2014 - "In previous clinical trials involving children with X-linked severe combined immunodeficiency (SCID-X1), a Moloney murine leukemia virus-based γ-retrovirus vector expressing interleukin-2 receptor γ-chain (γc) complementary DNA successfully restored immunity in most patients but resulted in vector-induced leukemia through enhancer-mediated mutagenesis in 25% of patients. "
06/01/1994 - "The complete nucleotide sequence of the cDNA indicates that it is related most closely to EMT, a tyrosine kinase of T cells and to the B-cell tyrosine kinase Btk, which is mutated in X-linked agammaglobulinemia (XLA) in humans and X-linked immunodeficiency disease (XID) in mouse. "
|7.||Interleukin-4 (Interleukin 4)IBA
03/01/2002 - "Several gamma(c) residues involved in IL-4 binding have been previously shown to be mutated in X-linked severe combined immunodeficiency. "
03/14/1997 - "Interleukin-4 signaling in B lymphocytes from patients with X-linked severe combined immunodeficiency."
03/01/1997 - "[The signal transduction mechanism of IL-4 in X-linked severe combined immunodeficiency patients]."
05/26/1994 - "10) and IL-4 (refs 11, 12) receptors, which when mutated results in X-linked severe combined immunodeficiency. "
01/15/1999 - "Functional role of interleukin-4 (IL-4) and IL-7 in the development of X-linked severe combined immunodeficiency."
|8.||Interleukin-15 (Interleukin 15)IBA
08/15/1995 - "Characterization of B-cell lines established from two X-linked severe combined immunodeficiency patients: interleukin-15 binds to the B cells but is not internalized efficiently."
05/01/1995 - "A defect in IL-15 signaling may therefore contribute to the phenotype of X-linked severe combined immunodeficiency in humans, resulting from mutations in the common gamma chain. "
01/01/1995 - "In this study, we have used X-linked severe combined immunodeficiency (X-SCID) as a naturally occurring gamma c-chain gene disruption model to examine the role of the gamma c-chain in human B-cell responses to IL-2, IL-4, IL-13, and IL-15. "
06/15/1994 - "Hence, IL-15, like IL-2, IL-4 and IL-7, utilizes the common IL-2R gamma subunit found to be defective in X-linked severe combined immunodeficiency in humans. "
11/22/2002 - "The cytokine interleukin-21 (IL-21) is closely related to IL-2 and IL-15, and their receptors all share the common cytokine receptor gamma chain, gammac, which is mutated in humans with X-linked severe combined immunodeficiency disease (XSCID). "
|10.||Prostaglandins D (PGD)IBA
12/18/2013 - "In the study, 12 PGD systems were set up including 6 different monogenic diseases (spinal muscular atrophy, Duchenne muscular dystrophy, X-linked chronic granulomatous disease, osteopetrosis, achondroplasia, X-linked severe combined immunodeficiency), Robertsonian translocations, α-thalassemia combined with Robertsonian translocation, α- and β-double thalassemia, β-thalassemia with HLA typing and DMD with HLA typing. "
|1.||Heterologous Transplantation (Xenotransplantation)
06/01/2011 - "Marking studies in humans, murine xenografts, and large animals have helped optimize conditions for gene transfer into CD34(+) hematopoietic progenitors, contributing to the achievement of gene transfer efficiencies sufficient for clinical benefit in several serious genetic diseases such as X-linked severe combined immunodeficiency and adrenoleukodystrophy. "
|2.||Stem Cell Transplantation
04/18/2002 - "X-linked severe combined immunodeficiency due to a mutation in the gene encoding the common gamma (gamma(c)) chain is a lethal condition that can be cured by allogeneic stem-cell transplantation. "
02/01/2008 - "Rituximab for lymphoproliferative disease prior to haematopoietic stem cell transplantation for X-linked severe combined immunodeficiency."
|3.||Cord Blood Stem Cell Transplantation
09/01/2002 - "The role of common gamma chain in human monocytes in vivo; evaluation from the studies of X-linked severe combined immunodeficiency (X-SCID) carriers and X-SCID patients who underwent cord blood stem cell transplantation."
09/01/2002 - "Expression of common gamma chain (gammac) on monocytes was studied in five carriers of X-linked severe combined immunodeficiency (X-SCID) and two X-SCID patients who underwent cord blood stem cell transplantation (CBSCT). "
03/01/2007 - "X-linked severe combined immunodeficiency (X-SCID) with high blood levels of immunoglobulins and Aspergillus pneumonia successfully treated with micafangin followed by unrelated cord blood stem cell transplantation."
01/01/2013 - "We have used two distinct mouse serial transplant assays to evaluate the safety of a self-inactivating lentiviral vector intended for use in X-linked severe combined immunodeficiency (XSCID) gene therapy trials. "
09/01/2007 - "Dogs with X-linked severe combined immunodeficiency (XSCID) can be successfully treated by bone marrow transplants (BMT) resulting in full immunologic reconstitution and engraftment of both donor B and T cells without the need for pretransplant conditioning. "
|5.||Transplantation (Transplant Recipients)
03/30/2015 - "X-linked severe combined immunodeficiency (X-SCID) rats for xeno-transplantation and behavioral evaluation."
06/15/2007 - "Marking of peripheral T-lymphocytes by retroviral transduction and transplantation of CD34+ cells in a canine X-linked severe combined immunodeficiency model."
08/01/2002 - "Prenatal T-cell reconstitution after in utero transplantation with fetal liver cells in a patient with X-linked severe combined immunodeficiency."
12/12/1996 - "Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow."
11/30/1996 - "In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI)."