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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

Nature Medicine, 2022-01, Vol.28 (1), p.71-80

2022

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Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Nature Medicine, 2022-10, Vol.28 (10), p.2217-2217

2022

Volltextzugriff (PDF)

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

Nature genetics, 2000-07, Vol.25 (3), p.306-310

2000

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Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells

Nature communications, 2017-11, Vol.8 (1), p.1576-13, Article 1576

2017

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Effective gene therapy with nonintegrating lentiviral vectors

Nature medicine, 2006-03, Vol.12 (3), p.348-353

2006

Volltextzugriff (PDF)

Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells

Nature communications, 2016-07, Vol.7 (1), p.12175-15, Article 12175

2016

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RISC control for gene therapy

Nature biotechnology, 2006-06, Vol.24 (6), p.661-662

2006

Volltextzugriff (PDF)

Ectopic retroviral expression of LMO2, but not IL2Rg, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy

Leukemia, 2007-04, Vol.21 (4), p.754-763

2007

Volltextzugriff (PDF)

Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy

Leukemia, 2007-04, Vol.21 (4), p.754

2007

Volltextzugriff (PDF)

Looks, reads, and feels

Library journal (1976), 2004-07, Vol.129 (12), p.12

Thrasher, Jerry A

2004

Volltextzugriff (PDF)

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Nature communications, 2020-08, Vol.11 (1), p.4034-15, Article 4034

2020

Volltextzugriff (PDF)

Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection

Nature communications, 2016-12, Vol.7 (1), p.13992-12, Article 13992

2016

Volltextzugriff (PDF)

Phosphoinositide 3-OH kinase p85α and p110β are essential for androgen receptor transactivation and tumor progression in prostate cancers

Oncogene, 2008-07, Vol.27 (33), p.4569-4579

2008

Volltextzugriff (PDF)

Platelet actin nodules are podosome-like structures dependent on Wiskott-Aldrich syndrome protein and ARP2/3 complex

Nature communications, 2015-06, Vol.6 (1), p.7254-7254, Article 7254

2015

Volltextzugriff (PDF)

A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion

Nature communications, 2021-07, Vol.12 (1), p.4358-4358, Article 4358

2021

Volltextzugriff (PDF)

Perceived barriers to the adoption of active surveillance in low-risk prostate cancer: a qualitative analysis of community and academic urologists

BMC cancer, 2021-05, Vol.21 (1), p.1-12, Article 649

2021

Volltextzugriff (PDF)

WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis

Nature communications, 2018-05, Vol.9 (1), p.1779-15, Article 1779

2018

Volltextzugriff (PDF)

Gene therapy matures in the clinic

Nature biotechnology, 2012-07, Vol.30 (7), p.588-593

2012

Volltextzugriff (PDF)

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

Nature communications, 2021-03, Vol.12 (1), p.1622-16, Article 1622

2021

Volltextzugriff (PDF)

Site- and allele-specific polycomb dysregulation in T-cell leukaemia

Nature communications, 2015-01, Vol.6 (1), p.6094-6094, Article 6094

2015

Volltextzugriff (PDF)

GWAS meta-analysis of intrahepatic cholestasis of pregnancy implicates multiple hepatic genes and regulatory elements

Nature communications, 2022-08, Vol.13 (1), p.4840-4840, Article 4840

2022

Volltextzugriff (PDF)

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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome

Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy

Lentiviral gene therapy for X-linked chronic granulomatous disease

Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells

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

Effective gene therapy with nonintegrating lentiviral vectors

Deletion of Wiskott-Aldrich syndrome protein triggers Rac2 activity and increased cross-presentation by dendritic cells

RISC control for gene therapy

Ectopic retroviral expression of LMO2, but not IL2Rg, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy

Ectopic retroviral expression of LMO2, but not IL2Rgamma, blocks human T-cell development from CD34+ cells: implications for leukemogenesis in gene therapy

Looks, reads, and feels

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection

Phosphoinositide 3-OH kinase p85α and p110β are essential for androgen receptor transactivation and tumor progression in prostate cancers

Platelet actin nodules are podosome-like structures dependent on Wiskott-Aldrich syndrome protein and ARP2/3 complex

A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion

Perceived barriers to the adoption of active surveillance in low-risk prostate cancer: a qualitative analysis of community and academic urologists

WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis

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

Gene therapy matures in the clinic

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

Comprehensive genomic access to vector integration in clinical gene therapy

Site- and allele-specific polycomb dysregulation in T-cell leukaemia

GWAS meta-analysis of intrahepatic cholestasis of pregnancy implicates multiple hepatic genes and regulatory elements

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