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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

Nature communications, 2022-03, Vol.13 (1), p.1315-13, Article 1315

2022

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A Comeback for Gene Therapy

Science (American Association for the Advancement of Science), 2009-11, Vol.326 (5954), p.805-806

Naldini, Luigi

2009

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Gene therapy returns to centre stage

Nature (London), 2015-10, Vol.526 (7573), p.351-360

Naldini, Luigi

2015

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Ensuring a future for gene therapy for rare diseases

Nature medicine, 2022-10, Vol.28 (10), p.1985-1988

2022

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Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

Nature biotechnology, 2020-11, Vol.38 (11), p.1298-1308

2020

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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

Nature reviews. Genetics, 2009-08, Vol.10 (8), p.578-585

2009

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Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

Cell stem cell, 2019-04, Vol.24 (4), p.551-565.e8

2019

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Adopt a moratorium on heritable genome editing

Nature (London), 2019-03, Vol.567 (7747), p.165-168

2019

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Ex vivo gene transfer and correction for cell-based therapies

Nature reviews. Genetics, 2011-05, Vol.12 (5), p.301-315

Naldini, Luigi

2011

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Targeting the ANG2/TIE2 Axis Inhibits Tumor Growth and Metastasis by Impairing Angiogenesis and Disabling Rebounds of Proangiogenic Myeloid Cells

Cancer cell, 2011-04, Vol.19 (4), p.512-526

2011

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Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Science translational medicine, 2017-10, Vol.9 (411)

2017

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Angiopoietin-2 TIEs Up Macrophages in Tumor Angiogenesis

Clinical cancer research, 2011-08, Vol.17 (16), p.5226-5232

2011

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Tie2-expressing monocytes and tumor angiogenesis : Regulation by hypoxia and angiopoietin-2

Cancer research (Chicago, Ill.), 2007-09, Vol.67 (18), p.8429-8432

2007

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BAR-Seq clonal tracking of gene-edited cells

Nature protocols, 2021-06, Vol.16 (6), p.2991-3025

2021

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In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

Cell stem cell, 2016-07, Vol.19 (1), p.107-119

2016

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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

The Journal of clinical investigation, 2009-04, Vol.119 (4), p.964-975

2009

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Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

The Journal of clinical investigation, 2012-05, Vol.122 (5), p.1667-1676

2012

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Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

Science translational medicine, 2019-05, Vol.11 (493)

2019

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Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

Nature communications, 2018-07, Vol.9 (1), p.2896-16, Article 2896

2018

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A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective antitumor responses in mice

Blood, 2013-07, Vol.122 (2), p.243-252

2013

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Retrieval of vector integration sites from cell-free DNA

Nature medicine, 2021-08, Vol.27 (8), p.1458-1470

2021

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Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

Proceedings of the National Academy of Sciences - PNAS, 2012-09, Vol.109 (37), p.15018-15023

2012

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

Nature biotechnology, 2007-11, Vol.25 (11), p.1298-1306

2007

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A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes, blood “resident” monocytes, and embryonic macrophages suggests common functions and developmental relationships

Blood, 2009-07, Vol.114 (4), p.901-914

2009

Volltextzugriff (PDF)

Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Nature communications, 2022-05, Vol.13 (1), p.2454-2454, Article 2454

2022

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A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders

A Comeback for Gene Therapy

Gene therapy returns to centre stage

Ensuring a future for gene therapy for rare diseases

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

Adopt a moratorium on heritable genome editing

Ex vivo gene transfer and correction for cell-based therapies

Targeting the ANG2/TIE2 Axis Inhibits Tumor Growth and Metastasis by Impairing Angiogenesis and Disabling Rebounds of Proangiogenic Myeloid Cells

Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1

Angiopoietin-2 TIEs Up Macrophages in Tumor Angiogenesis

Tie2-expressing monocytes and tumor angiogenesis : Regulation by hypoxia and angiopoietin-2

BAR-Seq clonal tracking of gene-edited cells

In Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution Phases

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

Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations

Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates

Interferon gene therapy reprograms the leukemia microenvironment inducing protective immunity to multiple tumor antigens

A role for miR-155 in enabling tumor-infiltrating innate immune cells to mount effective antitumor responses in mice

Retrieval of vector integration sites from cell-free DNA

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

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

A distinguishing gene signature shared by tumor-infiltrating Tie2-expressing monocytes, blood “resident” monocytes, and embryonic macrophages suggests common functions and developmental relationships

Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

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