(Q38516548)

3 September 2017

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title

Gene therapy for monogenic disorders of the bone marrow (English)

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3 September 2017

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Adrian J Thrasher

2

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author name string

Sujal Ghosh

series ordinal

1

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3 September 2017

H Bobby Gaspar

series ordinal

3

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3 September 2017

language of work or name

English

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publication date

5 June 2015

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British Journal of Haematology

3 September 2017

published in

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

3 September 2017

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Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

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Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

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Chronic granulomatous disease: overview and hematopoietic stem cell transplantation

21 January 2018

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Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial

21 January 2018

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Management of sickle cell disease from childhood through adulthood

21 January 2018

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Stem cell collection and gene transfer in Fanconi anemia

21 January 2018

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Clinical, functional, and genetic characterization of chronic granulomatous disease in 89 Turkish patients

21 January 2018

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Cytokines and immunodeficiency diseases: critical roles of the gamma(c)-dependent cytokines interleukins 2, 4, 7, 9, 15, and 21, and their signaling pathways

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Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity

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Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients

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Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice

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

21 January 2018

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Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models

21 January 2018

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

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Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

21 January 2018

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Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects

21 January 2018

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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

Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer

21 January 2018

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

21 January 2018

1 reference

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

21 January 2018

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Transplantation outcomes for severe combined immunodeficiency, 2000-2009.

21 January 2018

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Correction of sickle cell disease in transgenic mouse models by gene therapy

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A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction

21 January 2018

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Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin

21 January 2018

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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.

21 January 2018

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Consequences and management of iron overload in sickle cell disease

21 January 2018

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Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector

21 January 2018

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Targeted gene therapy and cell reprogramming in Fanconi anemia

21 January 2018

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SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

21 January 2018

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Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs

21 January 2018

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21 January 2018

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New insights into the biology of Wiskott-Aldrich syndrome (WAS).

21 January 2018

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WASP: a key immunological multitasker

21 January 2018

1 reference

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1.

21 January 2018

1 reference

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

21 January 2018

1 reference

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene

21 January 2018

1 reference

Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome

21 January 2018

1 reference

Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting.

21 January 2018

1 reference

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21 January 2018

1 reference

Mapping of DNase I-hypersensitive sites in the upstream DNA of human embryonic epsilon-globin gene in K562 leukemia cells

21 January 2018

1 reference

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

21 January 2018

1 reference

Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

21 January 2018

1 reference

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac

21 January 2018

1 reference

Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency

21 January 2018

1 reference

Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1111/BJH.13520

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