(Q37858878)

24 August 2017

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title

Ex vivo gene transfer and correction for cell-based therapies (English)

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24 August 2017

1

Luigi Naldini

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publication date

29 March 2011

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24 August 2017

published in

Nature Reviews Genetics

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24 August 2017

volume

12

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24 August 2017

issue

5

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24 August 2017

page(s)

301-315

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Hematopoietic stem-cell transplantation

24 August 2017

cites work

1 reference

Adoptive cell therapy: genetic modification to redirect effector cell specificity.

21 January 2018

1 reference

Adoptive T cell therapy of cancer

21 January 2018

1 reference

T cell receptor gene therapy for cancer

21 January 2018

1 reference

Engineering lymphocyte subsets: tools, trials and tribulations

21 January 2018

1 reference

Epithelial stem cells in corneal regeneration and epidermal gene therapy

21 January 2018

1 reference

Stem cells in human neurodegenerative disorders--time for clinical translation?

21 January 2018

1 reference

Erratum: Neural stem cell systems: physiological players or in vitro entities?

21 January 2018

1 reference

Cardiogenesis and the complex biology of regenerative cardiovascular medicine

21 January 2018

1 reference

Mesenchymal stem cells: mechanisms of inflammation

21 January 2018

1 reference

Mesenchymal stem cells: revisiting history, concepts, and assays.

21 January 2018

1 reference

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

Efficacy of gene therapy for X-linked severe combined immunodeficiency

21 January 2018

1 reference

20 years of gene therapy for SCID.

21 January 2018

1 reference

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

1 reference

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

21 January 2018

1 reference

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

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

1 reference

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

21 January 2018

1 reference

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

21 January 2018

1 reference

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

21 January 2018

1 reference

The expanding universe of transposon technologies for gene and cell engineering.

21 January 2018

1 reference

Genome editing with engineered zinc finger nucleases

21 January 2018

1 reference

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

1 reference

Bone marrow transplantation for primary immunodeficiency diseases

21 January 2018

1 reference

Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

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

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

21 January 2018

1 reference

Targeting gene-modified hematopoietic cells to the central nervous system: use of green fluorescent protein uncovers microglial engraftment.

21 January 2018

1 reference

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

Local self-renewal can sustain CNS microglia maintenance and function throughout adult life.

21 January 2018

1 reference

Microglia in the adult brain arise from Ly-6ChiCCR2+ monocytes only under defined host conditions

21 January 2018

1 reference

Genetically modified CD34+ hematopoietic stem cells contribute to turnover of brain perivascular macrophages in long-term repopulated primates.

21 January 2018

1 reference

Fate mapping analysis reveals that adult microglia derive from primitive macrophages

21 January 2018

1 reference

Gene therapy to protect haematopoietic cells from cytotoxic cancer drugs

21 January 2018

1 reference

Genetic therapies against HIV.

21 January 2018

1 reference

Platelets as delivery systems for disease treatments

21 January 2018

1 reference

Tumor-targeted interferon-alpha delivery by Tie2-expressing monocytes inhibits tumor growth and metastasis

21 January 2018

1 reference

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study.

21 January 2018

1 reference

Virus-specific T cells engineered to coexpress tumor-specific receptors: persistence and antitumor activity in individuals with neuroblastoma

21 January 2018

1 reference

Regulatory T-cell immunotherapy for tolerance to self antigens and alloantigens in humans.

21 January 2018

1 reference

Human T cell responses against melanoma

21 January 2018

1 reference

Adoptive transfer of effector CD8+ T cells derived from central memory cells establishes persistent T cell memory in primates

21 January 2018

1 reference

IL-7 and IL-15 allow the generation of suicide gene-modified alloreactive self-renewing central memory human T lymphocytes

21 January 2018

1 reference

Lethal graft-versus-host disease in mouse models of T cell receptor gene therapy

21 January 2018

1 reference

Mixed T cell receptor dimers harbor potentially harmful neoreactivity

21 January 2018

1 reference

Improved expression and reactivity of transduced tumor-specific TCRs in human lymphocytes by specific silencing of endogenous TCR.

21 January 2018

1 reference

Transplantation of mouse HSCs genetically modified to express a CD4-restricted TCR results in long-term immunity that destroys tumors and initiates spontaneous autoimmunity

21 January 2018

1 reference

Engineering human hematopoietic stem/progenitor cells to produce a broadly neutralizing anti-HIV antibody after in vitro maturation to human B lymphocytes

21 January 2018

1 reference

Engineering antigen-specific T cells from genetically modified human hematopoietic stem cells in immunodeficient mice

21 January 2018

1 reference

Regeneration next: toward heart stem cell therapeutics

21 January 2018

1 reference

Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

21 January 2018

1 reference

Repair of tissues by adult stem/progenitor cells (MSCs): controversies, myths, and changing paradigms

21 January 2018

1 reference

Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism.

21 January 2018

1 reference

Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease.

21 January 2018

1 reference

Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis

21 January 2018

1 reference

Communication via gap junctions underlies early functional and beneficial interactions between grafted neural stem cells and the host

21 January 2018

1 reference

Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease: a phase II study

21 January 2018

1 reference

Bone marrow and circulating stem/progenitor cells for regenerative cardiovascular therapy

21 January 2018

1 reference

Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment

21 January 2018

1 reference

Retrovirus-mediated gene transfer in primary T lymphocytes impairs their anti-Epstein-Barr virus potential through both culture-dependent and selection process-dependent mechanisms.

21 January 2018

1 reference

Immunologic potential of donor lymphocytes expressing a suicide gene for early immune reconstitution after hematopoietic T-cell-depleted stem cell transplantation

21 January 2018

1 reference

Acquisition of full effector function in vitro paradoxically impairs the in vivo antitumor efficacy of adoptively transferred CD8+ T cells

21 January 2018

1 reference

Prostaglandin E2 regulates vertebrate haematopoietic stem cell homeostasis

21 January 2018

1 reference

Angiopoietin-like 5 and IGFBP2 stimulate ex vivo expansion of human cord blood hematopoietic stem cells as assayed by NOD/SCID transplantation

21 January 2018

1 reference

Pleiotrophin regulates the expansion and regeneration of hematopoietic stem cells

21 January 2018

1 reference

Aryl Hydrocarbon Receptor Antagonists Promote the Expansion of Human Hematopoietic Stem Cells

21 January 2018

1 reference

Genome-wide analysis of retroviral DNA integration

21 January 2018

1 reference

Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells

21 January 2018

1 reference

Efficient stable gene transfer into human cells by the Sleeping Beauty transposon vectors.

21 January 2018

1 reference

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

21 January 2018

1 reference

The phiC31 integrase system for gene therapy

21 January 2018

1 reference

Site-specific integration of retroviral DNA in human cells using fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc-finger protein E2C.

21 January 2018

1 reference

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

21 January 2018

1 reference

Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells

21 January 2018

1 reference

Cytokine signals are sufficient for HIV-1 infection of resting human T lymphocytes

21 January 2018

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Cytokines, including stem cell factor alone, enhance lentiviral transduction in nondividing human LTCIC and NOD/SCID repopulating cells

21 January 2018

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Human T lymphocytes transduced by lentiviral vectors in the absence of TCR activation maintain an intact immune competence

21 January 2018

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Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction.

21 January 2018

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Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell

21 January 2018

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Insertional mutagenesis in gene therapy and stem cell biology

21 January 2018

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High-throughput insertional mutagenesis screens in mice to identify oncogenic networks

21 January 2018

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Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis.

21 January 2018

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

21 January 2018

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

21 January 2018

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The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

21 January 2018

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Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors

21 January 2018

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Physiological promoters reduce the genotoxic risk of integrating gene vectors

21 January 2018

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

21 January 2018

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Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells

21 January 2018

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

21 January 2018

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Retroviral vector integration deregulates gene expression but has no consequence on the biology and function of transplanted T cells.

21 January 2018

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Silencing and variegation of gammaretrovirus and lentivirus vectors

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

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Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

21 January 2018

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

21 January 2018

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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

21 January 2018

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Stable knockdown of microRNA in vivo by lentiviral vectors

21 January 2018

1 reference

Progress and prospects: zinc-finger nucleases as gene therapy agents

21 January 2018

1 reference

Selection-free zinc-finger-nuclease engineering by context-dependent assembly (CoDA)

21 January 2018

1 reference

Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy

21 January 2018

1 reference

Targeting DNA double-strand breaks with TAL effector nucleases

21 January 2018

1 reference

A TALE nuclease architecture for efficient genome editing

21 January 2018

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Chimeric nucleases stimulate gene targeting in human cells

21 January 2018

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The DNA Damage Response: Making It Safe to Play with Knives

21 January 2018

1 reference

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

21 January 2018

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Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo

21 January 2018

1 reference

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

1 reference

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

21 January 2018

1 reference

Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells

21 January 2018

1 reference

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

21 January 2018

1 reference

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

21 January 2018

1 reference

Removal of homeostatic cytokine sinks by lymphodepletion enhances the efficacy of adoptively transferred tumor-specific CD8+ T cells

21 January 2018

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Pluripotency and cellular reprogramming: facts, hypotheses, unresolved issues

21 January 2018

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Somatic mosaicism in primary immune deficiencies

21 January 2018

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In vivo selection of MGMT(P140K) lentivirus–transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning

21 January 2018

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Efficient and stable MGMT-mediated selection of long-term repopulating stem cells in nonhuman primates.

21 January 2018

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Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region

21 January 2018

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Comparison of human cord blood engraftment between immunocompromised mouse strains

21 January 2018

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Large animal models of hematopoietic stem cell gene therapy

21 January 2018

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Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia

21 January 2018

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Stable marking and transgene expression without progression to monoclonality in canine long-term hematopoietic repopulating cells transduced with lentiviral vectors

21 January 2018

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Restriction of retroviral replication by APOBEC3G/F and TRIM5alpha

21 January 2018

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

21 January 2018

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DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

21 January 2018

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

21 January 2018

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

21 January 2018

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High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion

21 January 2018

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Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking.

21 January 2018

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Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells

21 January 2018

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Retroviral vector insertion sites associated with dominant hematopoietic clones mark "stemness" pathways

21 January 2018

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Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate

21 January 2018

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Real-time definition of non-randomness in the distribution of genomic events

21 January 2018

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Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

21 January 2018

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Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis

21 January 2018

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NOD/SCID repopulating cells contribute only to short-term repopulation in the baboon

21 January 2018

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Comprehensive genomic access to vector integration in clinical gene therapy

21 January 2018

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

21 January 2018

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Gene therapy fulfilling its promise

21 January 2018

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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

21 January 2018

1 reference

Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients

21 January 2018

1 reference

RNA-based gene therapy for HIV with lentiviral vector-modified CD34(+) cells in patients undergoing transplantation for AIDS-related lymphoma

21 January 2018

1 reference

Limbal Stem-Cell Therapy and Long-Term Corneal Regeneration

21 January 2018

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Neural stem cell systems: physiological players or in vitro entities?

31 March 2020

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Primary immunodeficiencies

31 March 2020

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Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy

31 March 2020

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Natural innate and adaptive immunity to cancer

31 March 2020

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Refined human artificial chromosome vectors for gene therapy and animal transgenesis

31 March 2020

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Thrombopoietin, flt3, and kit ligands together suppress apoptosis of human mobilized CD34+ cells and recruit primitive CD34+ Thy-1+ cells into rapid division

31 March 2020

1 reference

Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16

31 March 2020

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Safety of retroviral gene marking with a truncated NGF receptor

31 March 2020

1 reference

Assays to evaluate the genotoxicity of retroviral vectors

31 March 2020

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Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials.

31 March 2020

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Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

31 March 2020

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Expansion of human cord blood CD34(+)CD38(-) cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: dissociation of SRC phenotype and function.

31 March 2020

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12 December 2020

Current prospects for RNA interference-based therapies

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12 December 2020

Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells

1 reference

12 December 2020

In vivo selection of hematopoietic progenitor cells and temozolomide dose intensification in rhesus macaques through lentiviral transduction with a drug resistance gene

1 reference

12 December 2020

State-of-the-art gene-based therapies: the road ahead

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12 December 2020

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

1 reference

12 December 2020

Identifiers

DOI

10.1038/NRG2985

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24 August 2017

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