Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE. (Q39413079)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24448160%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

title

Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE (English)

1 reference

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5 March 2020

0 references

1 reference

4

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5 March 2020

Ivano Di Meo

5

1 reference

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5 March 2020

Jordi Barquinero

6

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5 March 2020

Alberto Auricchio

7

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5 March 2020

Ramon Martí

11

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5 March 2020

10

Massimo Zeviani

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5 March 2020

2

Carlo Viscomi

1 reference

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5 March 2020

author name string

Javier Torres-Torronteras

1

1 reference

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5 March 2020

Raquel Cabrera-Pérez

3

1 reference

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5 March 2020

Giuseppe Pizzorno

8

1 reference

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5 March 2020

Michio Hirano

9

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5 March 2020

language of work or name

1 reference

22 January 2014

1 reference

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5 March 2020

number of pages

7

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based on heuristic

inferred from page(s)

published in

Molecular Therapy

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5 March 2020

volume

22

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5 March 2020

issue

5

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5 March 2020

page(s)

901-907

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Effective AAV-mediated gene therapy in a mouse model of ethylmalonic encephalopathy

5 March 2020

cites work

1 reference

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11 September 2017

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Limited dCTP availability accounts for mitochondrial DNA depletion in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)

11 September 2017

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11 September 2017

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

11 September 2017

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Unbalanced deoxynucleotide pools cause mitochondrial DNA instability in thymidine phosphorylase-deficient mice

11 September 2017

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Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

11 September 2017

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11 September 2017

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Thymidine and deoxyuridine accumulate in tissues of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)

11 September 2017

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Allogeneic stem cell transplantation corrects biochemical derangements in MNGIE

11 September 2017

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Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

11 September 2017

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Mammalian nucleoside transporters

11 September 2017

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

11 September 2017

1 reference

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Altered thymidine metabolism due to defects of thymidine phosphorylase

11 September 2017

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Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver

11 September 2017

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Thymidine phosphorylase gene mutations in MNGIE, a human mitochondrial disorder

11 September 2017

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A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding

11 September 2017

1 reference

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Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE.

11 September 2017

1 reference

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Gene therapy vectors based on adeno-associated virus type 1.

14 September 2017

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Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo

14 September 2017

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Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line.

25 September 2017

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Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration

25 September 2017

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21 August 2018

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21 August 2018

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21 August 2018

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21 August 2018

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21 August 2018

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21 August 2018

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Mitochondrial DNA Depletion and Thymidine Phosphate Pool Dynamics in a Cellular Model of Mitochondrial Neurogastrointestinal Encephalomyopathy

21 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24448160

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Infusion of platelets transiently reduces nucleoside overload in MNGIE

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/24448160

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2014.6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24448160%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24448160%20AND%20SRC:MED&resulttype=core&format=json

5 March 2020

PubMed publication ID

24448160

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:24448160%20AND%20SRC:MED&resulttype=core&format=json