Advances of gene therapy for primary immunodeficiencies (Q26742031)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

review article

1 reference

title

Advances of gene therapy for primary immunodeficiencies (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

0 references

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

author name string

Fabio Candotti

series ordinal

1

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language of work or name

English

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publication date

9 March 2016

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

2 references

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

volume

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

Creative Commons Attribution 4.0 International

20 March 2020

page(s)

310

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copyright license

start time

9 March 2016

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

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Successful Combination of Sequential Gene Therapy and Rescue Allo-HSCT in Two Children with X-CGD - Importance of Timing

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Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

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Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency

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A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

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Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

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Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity

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

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Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

28 September 2017

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

28 September 2017

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

28 September 2017

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Gene therapy for primary immunodeficiencies: Part 2.

28 September 2017

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

28 September 2017

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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

28 September 2017

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Chronic granulomatous disease: overview and hematopoietic stem cell transplantation

28 September 2017

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Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting

28 September 2017

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Gene Therapy for Canine Leukocyte Adhesion Deficiency with Lentiviral Vectors Using the Murine Stem Cell Virus and Human Phosphoglycerate Kinase Promoters

28 September 2017

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A tissue-specific, activation-inducible, lentiviral vector regulated by human CD40L proximal promoter sequences

28 September 2017

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Stem-cell gene therapy for the Wiskott-Aldrich syndrome

28 September 2017

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Gene therapy of chronic granulomatous disease: the engraftment dilemma

28 September 2017

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

28 September 2017

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Gene therapy of canine leukocyte adhesion deficiency using lentiviral vectors with human CD11b and CD18 promoters driving canine CD18 expression.

28 September 2017

1 reference

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

28 September 2017

1 reference

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Lentiviral vectors incorporating a human elongation factor 1alpha promoter for the treatment of canine leukocyte adhesion deficiency

28 September 2017

1 reference

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

28 September 2017

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B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia

28 September 2017

1 reference

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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

28 September 2017

1 reference

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How I treat ADA deficiency

28 September 2017

1 reference

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Restoration of NET formation by gene therapy in CGD controls aspergillosis

28 September 2017

1 reference

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Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

28 September 2017

1 reference

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Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells.

28 September 2017

1 reference

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

28 September 2017

1 reference

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Development of an enhanced B-specific lentiviral vector expressing BTK: a tool for gene therapy of XLA.

28 September 2017

1 reference

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Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

28 September 2017

1 reference

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Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy.

28 September 2017

1 reference

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

28 September 2017

1 reference

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Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction.

28 September 2017

1 reference

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Gene therapy for leukocyte adhesion deficiency.

28 September 2017

1 reference

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Gene-transferred oligoclonal T cells predominantly persist in peripheral blood from an adenosine deaminase-deficient patient during gene therapy

28 September 2017

1 reference

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Virus-specific immunity after gene therapy in a murine model of severe combined immunodeficiency

28 September 2017

1 reference

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Construction of retroviral vectors carrying human CD3 gamma cDNA and reconstitution of CD3 gamma expression and T cell receptor surface expression and function in a CD3 gamma-deficient mutant T cell line

28 September 2017

1 reference

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Signaling via IL-2 and IL-4 in JAK3-deficient severe combined immunodeficiency lymphocytes: JAK3-dependent and independent pathways.

28 September 2017

1 reference

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Reconstitution of T cell receptor signaling in ZAP-70-deficient cells by retroviral transduction of the ZAP-70 gene.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

In vitro correction of JAK3-deficient severe combined immunodeficiency by retroviral-mediated gene transduction

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

The interleukin-2 receptor gamma chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, SCIDX1.

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Correction of adenosine deaminase deficiency in cultured human T and B cells by retrovirus-mediated gene transfer

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Improved transfer of the leukocyte integrin CD18 subunit into hematopoietic cell lines by using retroviral vectors having a gibbon ape leukemia virus envelope.

28 September 2017

1 reference

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Targeted genome editing in human repopulating haematopoietic stem cells

28 September 2017

1 reference

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

31 May 2018

1 reference

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

31 May 2018

1 reference

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Correction of defective expression in MHC class II deficiency (bare lymphocyte syndrome) cells by retroviral transduction of CIITA

31 May 2018

1 reference

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Bone marrow gene transfer in three patients with adenosine deaminase deficiency.

31 May 2018

1 reference

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

31 May 2018

1 reference

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Correction of proliferative responses in purine nucleoside phosphorylase (PNP)-deficient T lymphocytes by retroviral-mediated PNP gene transfer and expression

31 May 2018

1 reference

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

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Correction of CD18-deficient lymphocytes by retrovirus-mediated gene transfer

31 May 2018

1 reference

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Partial phenotypic correction of human Lesch-Nyhan (hypoxanthine-guanine phosphoribosyltransferase-deficient) lymphoblasts with a transmissible retroviral vector

31 May 2018

1 reference

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Successful RAG1-SCID gene therapy depends on the level of RAG1 expression.

31 May 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Lentivirus gene therapy for purine nucleoside phosphorylase deficiency

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Stable and functional lymphoid reconstitution in artemis-deficient mice following lentiviral artemis gene transfer into hematopoietic stem cells.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Retrovirus-mediated transduction of primary ZAP-70-deficient human T cells results in the selective growth advantage of gene-corrected cells: implications for gene therapy

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Thymic lymphoproliferative disease after successful correction of CD40 ligand deficiency by gene transfer in mice

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Retroviral-mediated gene transfer of the leukocyte integrin CD18 into peripheral blood CD34 cells derived from a patient with leukocyte adhesion deficiency type 1

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Retroviral mediated expression of CD18 in normal and deficient human bone marrow progenitor cells

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

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

28 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4963077

Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor

28 November 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27508076

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

1 reference

https://pubmed.ncbi.nlm.nih.gov/27508076

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Gene therapy for chronic granulomatous disease

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27508076

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.12688/F1000RESEARCH.7512.1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27508076 AND SRC:MED&resulttype=core&format=json

20 March 2020

PubMed publication ID

27508076

1 reference