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Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

Human gene therapy, 2017-01, Vol.28 (1), p.112

2017

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Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

Blood, 2012-11, Vol.120 (18), p.3677-3687

2012

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Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Blood, 2017-05, Vol.129 (19), p.2624-2635

2017

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Clonality analysis after retroviral-mediated gene transfer to CD34 + cells from the cord blood of ADA-deficient SCID neonates

Nature medicine, 2003-04, Vol.9 (4), p.463-468

2003

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Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

Molecular therapy. Methods & clinical development, 2021-03, Vol.20, p.765-778

2021

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Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Molecular therapy. Methods & clinical development, 2020-03, Vol.16, p.78-93

2020

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Letter to the Editor. Differences between lentiviral vector-mediated ex vivo hematopoietic stem cell and adeno-associated virus gene therapies for neurological disorders

Journal of neurosurgery, 2021-12, Vol.136 (5), p.1501-1501

2021

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Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations

Journal of clinical immunology, 2017-10, Vol.37 (7), p.626-637

2017

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Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34 cells

Molecular therapy, 2005-07, Vol.12 (1), p.77-86

2005

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Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Blood, 2008-06, Vol.111 (12), p.5745-5754

2008

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Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

Blood, 2007-01, Vol.109 (2), p.503-506

2007

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1090. Enzyme Replacement Therapy with Pegylated Adenosine Deaminase (PEG-ADA) Does Not Impede Immune Reconstitution Following Transplantation of Gene-Corrected Bone Marrow Cells in the Murine Model of ADA-SCID

Molecular therapy, 2006-05, Vol.13 (S1), p.S419-S419

2006

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Expression from Second-Generation Feline Immunodeficiency Virus Vectors Is Impaired in Human Hematopoietic Cells

Molecular therapy, 2002-11, Vol.6 (5), p.645-652

2002

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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

The New England journal of medicine, 2021-05, Vol.384 (21), p.2002-2013

2021

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Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency

Biology of blood and marrow transplantation, 2015-02, Vol.21 (2), p.S102-S102

2015

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C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency

Molecular therapy, 2015-05, Vol.23, p.S102-S103

2015

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A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34+ cells from the bone marrow of human immunodeficiency virus-1-infected children

Blood, 1999-07, Vol.94 (1), p.368-371

1999

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442. Decreased Survival and Engraftment in Adult ADA-Deficient Mice Co-Transplanted With Uncorrected Lineage-Positive Bone Marrow Cells Combined With Gene-Corrected Lineage-Negative Bone Marrow Cells

Molecular therapy, 2015-05, Vol.23, p.S175-S175

2015

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764. Dosing and Re-Administration of Intravenous Lentiviral Vector for Liver-Directed Gene Transfer in Young Rhesus Monkeys and ADA-Deficient Mice

Molecular therapy, 2016-05, Vol.24, p.S302-S303

2016

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The 24-hour time budget of Przewalski horses

Applied animal behaviour science, 1988, Vol.21 (1), p.5-17

1988

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1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in γc Gene Knock-Out Mice

Molecular therapy, 2006-05, Vol.13 (S1), p.S418-S418

2006

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1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in [gamma]c Gene Knock-Out Mice

Molecular therapy, 2006-05, Vol.13 (S1), p.S418

2006

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Expression from Second-Generation Feline Immunodeficiency Virus Vectors Is Impaired in Human Hematopoietic Cells

Molecular therapy, 2002-11, Vol.6 (5), p.645-652

2002

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Severe Combined Immunodeficiency - Therapy
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Adenosine Deaminase - Deficiency
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Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

Preclinical Demonstration of Lentiviral Vector-mediated Correction of Immunological and Metabolic Abnormalities in Models of Adenosine Deaminase Deficiency

Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients

Clonality analysis after retroviral-mediated gene transfer to CD34 + cells from the cord blood of ADA-deficient SCID neonates

Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

Letter to the Editor. Differences between lentiviral vector-mediated ex vivo hematopoietic stem cell and adeno-associated virus gene therapies for neurological disorders

Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID): Molecular Pathogenesis and Clinical Manifestations

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34 cells

Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

1090. Enzyme Replacement Therapy with Pegylated Adenosine Deaminase (PEG-ADA) Does Not Impede Immune Reconstitution Following Transplantation of Gene-Corrected Bone Marrow Cells in the Murine Model of ADA-SCID

Expression from Second-Generation Feline Immunodeficiency Virus Vectors Is Impaired in Human Hematopoietic Cells

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency

C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency

A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34+ cells from the bone marrow of human immunodeficiency virus-1-infected children

442. Decreased Survival and Engraftment in Adult ADA-Deficient Mice Co-Transplanted With Uncorrected Lineage-Positive Bone Marrow Cells Combined With Gene-Corrected Lineage-Negative Bone Marrow Cells

764. Dosing and Re-Administration of Intravenous Lentiviral Vector for Liver-Directed Gene Transfer in Young Rhesus Monkeys and ADA-Deficient Mice

The 24-hour time budget of Przewalski horses

1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in γc Gene Knock-Out Mice

1089. Neonatal BMT of ADA-Deficient SCID Mice Results in Phenotypic Correction Despite Low Levels of Engraftment and Absence of the Selective T Lymphoid Expansion Seen in [gamma]c Gene Knock-Out Mice

Expression from Second-Generation Feline Immunodeficiency Virus Vectors Is Impaired in Human Hematopoietic Cells

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