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Gene therapy for primary immunodeficiencies
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scholarly article
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
title
Gene therapy for primary immunodeficiencies
(English)
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
main subject
gene therapy
0 references
author
Adrian Thrasher
series ordinal
4
1 reference
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
author name string
Christine Rivat
series ordinal
1
2 references
stated in
Crossref
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
Giorgia Santilli
series ordinal
2
2 references
stated in
Crossref
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
H Bobby Gaspar
series ordinal
3
1 reference
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
language of work or name
English
1 reference
stated in
PubMed
publication date
1 July 2012
1 reference
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
published in
Human Gene Therapy
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
volume
23
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
issue
7
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
page(s)
668-675
1 reference
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
cites work
Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
21 March 2017
Efficacy of gene therapy for X-linked severe combined immunodeficiency
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
21 March 2017
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
21 March 2017
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
7 April 2017
Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
7 April 2017
Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activity
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndrome
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Retroviral gene therapy for X-linked chronic granulomatous disease: results from phase I/II trial
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Stem-cell gene therapy for the Wiskott-Aldrich syndrome
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Gene therapy of chronic granulomatous disease: the engraftment dilemma
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Biochemical correction of X-CGD by a novel chimeric promoter regulating high levels of transgene expression in myeloid cells
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Restoration of NET formation by gene therapy in CGD controls aspergillosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Restriction of transgene expression to the B-lymphoid progeny of human lentivirally transduced CD34+ cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Epidermal Langerhans cell-targeted gene expression by a dectin-2 promoter
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Gene therapy for leukocyte adhesion deficiency.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 September 2017
Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
31 May 2018
Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
31 May 2018
Retrovirus and lentivirus vector design and methods of cell conditioning
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Failure of SCID-X1 gene therapy in older patients
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Specific transgene expression in human and mouse CD4+ cells using lentiviral vectors with regulatory sequences from the CD4 gene
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Lymphoid abnormalities in CD40 ligand transgenic mice suggest the need for tight regulation in gene therapy approaches to hyper immunoglobulin M (IgM) syndrome
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3404418
retrieved
28 November 2018
Identifiers
DOI
10.1089/HUM.2012.116
1 reference
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
PMC publication ID
3404418
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
PubMed publication ID
22691036
2 references
stated in
PubMed
stated in
Europe PubMed Central
PMC publication ID
3404418
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:22691036%20AND%20SRC:MED&resulttype=core&format=json
retrieved
4 February 2020
ResearchGate publication ID
225299898
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