A look to future directions in gene therapy research for monogenic diseases (Q33258821)

24 July 2017

6 references

22 April 2019

22 June 2019

25 June 2019

19 September 2019

29 October 2020

3 November 2020

A look to future directions in gene therapy research for monogenic diseases (English)

1 reference

biomedical investigative technique

24 July 2017

1 reference

29 October 2020

4 references

22 April 2019

22 June 2019

25 June 2019

19 September 2019

1 reference

19 September 2019

1 reference

3 November 2020

Matthew H Porteus

1

1 reference

24 July 2017

Jon P Connelly

series ordinal

2

1 reference

24 July 2017

Shondra M Pruett

series ordinal

3

1 reference

24 July 2017

1 September 2006

1 reference

24 July 2017

29 September 2006

6 references

22 April 2019

22 June 2019

25 June 2019

19 September 2019

29 October 2020

3 November 2020

7 references

24 July 2017

22 April 2019

22 June 2019

25 June 2019

19 September 2019

29 October 2020

3 November 2020

2

1 reference

24 July 2017

9

1 reference

24 July 2017

e133

1 reference

Creative Commons Attribution 4.0 International

24 July 2017

6 references

22 April 2019

22 June 2019

25 June 2019

19 September 2019

29 October 2020

on focus list of Wikimedia project

3 November 2020

copyright status

copyrighted

0 references

ScienceSource

0 references

cites work

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Gene therapy: therapeutic gene causing lymphoma

14 July 2018

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

14 July 2018

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Phage phiC31 integrase-mediated genomic integration of the common cytokine receptor gamma chain in human T-cell lines

14 July 2018

1 reference

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

14 July 2018

1 reference

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Efficient gene targeting in Drosophila with zinc-finger nucleases

14 July 2018

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

14 July 2018

1 reference

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Integration specificity of phage phiC31 integrase in the human genome

14 July 2018

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Viral gene therapy strategies: from basic science to clinical application

14 July 2018

1 reference

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Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells

14 July 2018

1 reference

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Genome-wide analysis of retroviral DNA integration

14 July 2018

1 reference

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Large-scale analysis of adeno-associated virus vector integration sites in normal human cells

14 July 2018

1 reference

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Sleeping beauty transposon-mediated gene therapy for prolonged expression

14 July 2018

1 reference

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Site-specific integration with phiC31 integrase for prolonged expression of therapeutic genes

14 July 2018

1 reference

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Evasion of the p53 tumour surveillance network by tumour-derived MYC mutants

14 July 2018

1 reference

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Gene therapy: twenty-first century medicine

14 July 2018

1 reference

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Gene targeting with viral vectors

14 July 2018

1 reference

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Drug therapy for acute myeloid leukemia

14 July 2018

1 reference

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

14 July 2018

1 reference

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Gene therapy for severe combined immunodeficiency

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Gene targeting in stem cells from individuals with osteogenesis imperfecta

14 July 2018

1 reference

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Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

14 July 2018

1 reference

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Chimeric nucleases stimulate gene targeting in human cells

14 July 2018

1 reference

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Progress and problems with the use of viral vectors for gene therapy

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Human Gene Targeting by Adeno-Associated Virus Vectors Is Enhanced by DNA Double-Strand Breaks

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Prenatal origin of TEL-AML1-positive acute lymphoblastic leukemia in children born in California

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Presence of clone-specific markers at birth in children with acute lymphoblastic leukaemia

14 July 2018

1 reference

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

14 July 2018

1 reference

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Development of virus vectors for gene therapy of beta chain hemoglobinopathies: flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo

14 July 2018

1 reference

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Modelling the molecular circuitry of cancer

14 July 2018

1 reference

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Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy

14 July 2018

1 reference

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Insulators: many functions, many mechanisms.

14 July 2018

1 reference

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Directed evolution of a recombinase for improved genomic integration at a native human sequence

14 July 2018

1 reference

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A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency

14 July 2018

1 reference

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A chromatin insulator protects retrovirus vectors from chromosomal position effects

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

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

14 July 2018

1 reference

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Human gene targeting by viral vectors

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Genetic manipulation of genomes with rare-cutting endonucleases

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Lessons from hereditary colorectal cancer

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

The two "hit" and multiple "hit" theories of carcinogenesis

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Mutation and cancer: statistical study of retinoblastoma

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Positive genetic selection for gene disruption in mammalian cells by homologous recombination

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

14 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Gene therapy insertional mutagenesis insights

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Stable nonviral genetic correction of inherited human skin disease

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

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

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Chromosomal effects of adeno-associated virus vector integration

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Limiting science: a biologist's perspective.

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

Origin and Function of Avian Retrovirus Transforming Genes

31 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1584267

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

31 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/17009872

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Report from the 2nd Stem Cell Clonality and Genotoxicity Retreat: St. Louis, MO, June 1, 2005

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/17009872

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1371/JOURNAL.PGEN.0020133

1 reference

release_yyrb3i3isfhlnddkys7md5tuly

24 July 2017

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/yyrb3i3isfhlnddkys7md5tuly

mapped directly with Wikidata item

24 November 2022

based on heuristic

PMCID

1584267

1 reference

24 July 2017

1 reference