Site-specific integration and tailoring of cassette design for sustainable gene transfer. (Q45872654)

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11 February 2020

title

Site-specific integration and tailoring of cassette design for sustainable gene transfer (English)

1 reference

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11 February 2020

author

Angelo Leone Lombardo

1

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11 February 2020

Bruno Di Stefano

4

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11 February 2020

Alessio Cantore

9

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11 February 2020

Daniela Cesana

2

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11 February 2020

Pietro Genovese

3

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11 February 2020

Daniele F Colombo

6

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11 February 2020

Pietro Lo Riso

10

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11 February 2020

Philip D Gregory

14

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11 February 2020

Angela Gritti

15

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11 February 2020

Vania Broccoli

16

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11 February 2020

Maria Chiara Bonini

17

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11 February 2020

Luigi Naldini

18

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11 February 2020

author name string

Elena Provasi

5

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11 February 2020

Margherita Neri

7

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11 February 2020

Zulma Magnani

8

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11 February 2020

Martina Damo

11

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11 February 2020

Oscar M Pello

12

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11 February 2020

Michael C Holmes

13

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11 February 2020

publication date

21 August 2011

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11 February 2020

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11 February 2020

volume

8

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11 February 2020

issue

10

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11 February 2020

page(s)

861-869

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Silencing and variegation of gammaretrovirus and lentivirus vectors

11 February 2020

cites work

1 reference

Ex vivo gene transfer and correction for cell-based therapies

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

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

21 January 2018

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Rapid "open-source" engineering of customized zinc-finger nucleases for highly efficient gene modification

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Chimeric nucleases stimulate gene targeting in human cells

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

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Genome editing with engineered zinc finger nucleases

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Next-generation gene targeting in the mouse for functional genomics

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Human genetic variation and its contribution to complex traits

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More than a decade of developmental gene expression atlases: where are we now?

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

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

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Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection

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Robust, persistent transgene expression in human embryonic stem cells is achieved with AAVS1-targeted integration

21 January 2018

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Site-specific integration of adeno-associated virus involves partial duplication of the target locus.

21 January 2018

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

21 January 2018

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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

21 January 2018

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Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

21 January 2018

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Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo

21 January 2018

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Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

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Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA.

21 January 2018

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A small regulatory element from chromosome 19 enhances liver-specific gene expression

21 January 2018

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Charting histone modifications and the functional organization of mammalian genomes

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

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Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells

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The complex eukaryotic transcriptome: unexpected pervasive transcription and novel small RNAs

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Induction of pluripotent stem cells from adult human fibroblasts by defined factors

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An improved zinc-finger nuclease architecture for highly specific genome editing

21 January 2018

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Suicide gene therapy of graft-versus-host disease induced by central memory human T lymphocytes

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

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The use of anti-CD3 and anti-CD28 monoclonal antibodies to clone and expand human antigen-specific T cells

21 January 2018

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Isolation and cloning of multipotential stem cells from the embryonic human CNS and establishment of transplantable human neural stem cell lines by epigenetic stimulation

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Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo

21 January 2018

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Efficient in vitro labeling of human neural precursor cells with superparamagnetic iron oxide particles: relevance for in vivo cell tracking

21 January 2018

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A ROCK inhibitor permits survival of dissociated human embryonic stem cells

21 January 2018

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Functional engraftment of human ES cell-derived dopaminergic neurons enriched by coculture with telomerase-immortalized midbrain astrocytes.

21 January 2018

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A new mathematical model for relative quantification in real-time RT-PCR

21 January 2018

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Comprehensive algorithm for quantitative real-time polymerase chain reaction

21 January 2018

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qBase relative quantification framework and software for management and automated analysis of real-time quantitative PCR data

21 January 2018

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Chromatin immunoprecipitation and microarray-based analysis of protein location

21 January 2018

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Physical Isolation of Nascent RNA Chains Transcribed by RNA Polymerase II: Evidence for Cotranscriptional Splicing

21 January 2018

1 reference

Chromatin remodeling and extragenic transcription at the MHC class II locus control region.

21 January 2018

1 reference

Targeted integration of adeno-associated virus (AAV) into human chromosome 19

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21857672

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21857672

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/NMETH.1674

1 reference

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Dimensions Publication ID

11 February 2020

1043058765

0 references

1 reference