(Q37191861)

18 August 2017

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title

Gene therapy of inherited diseases (English)

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18 August 2017

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1

Alain Fischer

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Marina Cavazzana

series ordinal

2

object named as

Marina Cavazzana-Calvo

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language of work or name

English

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publication date

1 June 2008

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18 August 2017

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18 August 2017

volume

371

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18 August 2017

issue

9629

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18 August 2017

page(s)

2044-2047

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Getting ready for gene-based medicine

18 August 2017

cites work

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Genetic medicines: treatment strategies for hereditary disorders

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Activation by locus control regions?

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Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs

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7 January 2021

RNA based gene therapy for dominantly inherited diseases

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Molecular biology. RNAi safety comes under scrutiny

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Gene targeting using zinc finger nucleases

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7 January 2021

Gene therapy: twenty-first century medicine

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7 January 2021

Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics

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7 January 2021

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

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7 January 2021

Transcription start regions in the human genome are favored targets for MLV integration

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7 January 2021

Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences

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7 January 2021

Retroviral integration and human gene therapy

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7 January 2021

HIV-1 integration in the human genome favors active genes and local hotspots

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7 January 2021

Distinct genomic integration of MLV and SIV vectors in primate hematopoietic stem and progenitor cells

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Murine leukemia induced by retroviral gene marking

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Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque

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7 January 2021

Site-specific genomic integration produces therapeutic Factor IX levels in mice

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7 January 2021

In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery.

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7 January 2021

CD8(+) T-cell responses to adeno-associated virus capsid in humans

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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7 January 2021

Effective gene therapy with nonintegrating lentiviral vectors

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Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

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7 January 2021

Effect of gene therapy on visual function in Leber's congenital amaurosis

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Safety and efficacy of gene transfer for Leber's congenital amaurosis

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7 January 2021

Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success

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7 January 2021

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

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7 January 2021

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells.

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7 January 2021

High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

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7 January 2021

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

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7 January 2021

Gene therapy for severe combined immunodeficiency

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7 January 2021

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

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7 January 2021

Gene therapy insertional mutagenesis insights

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7 January 2021

Gene therapy: therapeutic gene causing lymphoma

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7 January 2021

Gene therapy: X-SCID transgene leukaemogenicity

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7 January 2021

Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity

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7 January 2021

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

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7 January 2021

Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells

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7 January 2021

Physiological promoters reduce the genotoxic risk of integrating gene vectors

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7 January 2021

Prospects and implications of using chromatin insulators in gene therapy and transgenesis

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7 January 2021

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy

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7 January 2021

Gene therapy for Wiskott-Aldrich syndrome: rescue of T-cell signaling and amelioration of colitis upon transplantation of retrovirally transduced hematopoietic stem cells in mice

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Efficient transplantation via antibody-based clearance of hematopoietic stem cell niches

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

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7 January 2021

Development of gene therapy for blood disorders

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7 January 2021

10.1016/S0140-6736(08)60874-0

Identifiers

DOI

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18 August 2017

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