Lentiviral vectors for the treatment of primary immunodeficiencies (Q38195200)

30 August 2017

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title

Lentiviral vectors for the treatment of primary immunodeficiencies (English)

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30 August 2017

4

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

author name string

Giada Farinelli

series ordinal

1

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

Valentina Capo

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

Samantha Scaramuzza

series ordinal

3

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

language of work or name

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12 March 2014

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Journal of Inherited Metabolic Disease

30 August 2017

published in

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

volume

37

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

issue

4

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

9 June 2020

page(s)

525-533

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24619149 AND SRC:MED&resulttype=core&format=json

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

9 June 2020

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Restoration of NET formation by gene therapy in CGD controls aspergillosis

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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

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HIV integration site distributions in resting and activated CD4 T cells infected in culture

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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

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Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

21 January 2018

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Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.

21 January 2018

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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

21 January 2018

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Preclinical demonstration of lentiviral vector-mediated correction of immunological and metabolic abnormalities in models of adenosine deaminase deficiency

21 January 2018

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

21 January 2018

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Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.

21 January 2018

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Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency

21 January 2018

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Challenges in vector and trial design using retroviral vectors for long-term gene correction in hematopoietic stem cell gene therapy

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Molecular characterization of a large cohort of patients with Chronic Granulomatous Disease and identification of novel CYBB mutations: an Italian multicenter study

21 January 2018

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Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.

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Gene therapy of inherited diseases

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Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better?

21 January 2018

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Transduction of human CD34 repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.

21 January 2018

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Gene therapy of chronic granulomatous disease: the engraftment dilemma

21 January 2018

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21 January 2018

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Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study

21 January 2018

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A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

21 January 2018

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Efficacy of gene therapy for X-linked severe combined immunodeficiency

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Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency.

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Treatment of hemophagocytic lymphohistiocytosis with HLH-94 immunochemotherapy and bone marrow transplantation

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Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning

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Chronic granulomatous disease: overview and hematopoietic stem cell transplantation

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Gene therapy on the move

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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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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.

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21 January 2018

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Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

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

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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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21 January 2018

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Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment

21 January 2018

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CD4⁺ T cells from IPEX patients convert into functional and stable regulatory T cells by FOXP3 gene transfer

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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The repair of DNA damages/modifications during the maturation of the immune system: lessons from human primary immunodeficiency disorders and animal models.

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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Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells

21 January 2018

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Preclinical safety and efficacy of human CD34( ) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome

21 January 2018

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RAG mutations in human B cell-negative SCID.

21 January 2018

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A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of gammaC gene oncogenicity

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

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Crucial role of FOXP3 in the development and function of human CD25 CD4 regulatory T cells

21 January 2018

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Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells

21 January 2018

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21 January 2018

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A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells

21 January 2018

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A consensus document on the role of breakfast in the attainment and maintenance of health and wellness

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12 December 2020

Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy

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12 December 2020

Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency

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12 December 2020

Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter.

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12 December 2020

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

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12 December 2020