(Q55982397)

Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency.

21 January 2018

1 reference

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

21 January 2018

1 reference

Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

21 January 2018

1 reference

Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

21 January 2018

1 reference

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

1 reference

Cloning of human adenosine deaminase cDNA and expression in mouse cells

21 January 2018

1 reference

An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency

21 January 2018

1 reference

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

21 January 2018

1 reference

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

21 January 2018

1 reference

Adenosine deaminase-deficient mice generated using a two-stage genetic engineering strategy exhibit a combined immunodeficiency

21 January 2018

1 reference

Leukocyte adhesion deficiency: an inherited defect in the Mac-1, LFA-1, and p150,95 glycoproteins

21 January 2018

1 reference

Genetic, biochemical, and clinical features of chronic granulomatous disease.

21 January 2018

1 reference

Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

21 January 2018

1 reference

Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells

21 January 2018

1 reference

Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans

21 January 2018

1 reference

Mutations of Jak-3 gene in patients with autosomal severe combined immune deficiency (SCID)

21 January 2018

1 reference

Mutation of Jak3 in a patient with SCID: essential role of Jak3 in lymphoid development.

21 January 2018

1 reference

Defective IL7R expression in T(-)B(+)NK(+) severe combined immunodeficiency

21 January 2018

1 reference

Cytokines and immunodeficiency diseases

21 January 2018

1 reference

Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor.

21 January 2018

1 reference

Restoration of lymphocyte function in Janus kinase 3-deficient mice by retroviral-mediated gene transfer

21 January 2018

1 reference

Self-selection by genetically modified committed lymphocyte precursors reverses the phenotype of JAK3-deficient mice without myeloablation

21 January 2018

1 reference

Lymphoid development and function in X-linked severe combined immunodeficiency mice after stem cell gene therapy

21 January 2018

1 reference

Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy

21 January 2018

1 reference

Identification of clonogenic common lymphoid progenitors in mouse bone marrow

21 January 2018

1 reference

Identification of a novel, human multilymphoid progenitor in cord blood

21 January 2018

1 reference

RAG mutations in human B cell-negative SCID.

21 January 2018

1 reference

Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency.

21 January 2018

1 reference

Somatic mosaicism in Wiskott--Aldrich syndrome suggests in vivo reversion by a DNA slippage mechanism.

21 January 2018

1 reference

The Wiskott-Aldrich syndrome protein (WASP): roles in signaling and cytoskeletal organization

21 January 2018

1 reference

Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1

21 January 2018

1 reference

HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

21 January 2018

1 reference

Preselective gene therapy for Fabry disease

21 January 2018

1 reference

Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

21 January 2018

1 reference

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

21 January 2018

1 reference

Use of Serum-Free Medium with Fibronectin Fragment Enhanced Transduction in a System of Gas Permeable Plastic Containers to Achieve High Levels of Retrovirus Transduction at Clinical Scale

21 January 2018

1 reference

Thymic function after hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency

21 January 2018

1 reference

Adenosine-deaminase deficiency in two patients with severely impaired cellular immunity

31 March 2020

1 reference

Atypical X-Linked Severe Combined Immunodeficiency Due to Possible Spontaneous Reversion of the Genetic Defect in T Cells

31 March 2020

1 reference

Spontaneous in vivo reversion to normal of an inherited mutation in a patient with adenosine deaminase deficiency

31 March 2020

1 reference

Inherited Neutrophil Disorders: Molecular Basis and New Therapies

31 March 2020

1 reference