Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE. (Q52568126)

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24 March 2020

title

Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE (English)

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24 March 2020

14

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Ramon Martí

16

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author name string

Rana Yadak

1

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24 March 2020

Raquel Cabrera-Pérez

2

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24 March 2020

Javier Torres-Torronteras

3

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24 March 2020

Marianna Bugiani

4

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Joost C Haeck

5

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24 March 2020

Marshall W Huston

6

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Elly Bogaerts

7

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Steffi Goffart

8

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Edwin H Jacobs

9

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Merel Stok

10

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24 March 2020

Lorena Leonardelli

11

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24 March 2020

Luca Biasco

12

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Robert M Verdijk

13

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24 March 2020

George Ruijter

15

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24 March 2020

Gerard Wagemaker

17

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24 March 2020

Niek P van Til

18

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Irenaeus F M de Coo

19

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24 March 2020

publication date

8 January 2018

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Molecular Therapy. Methods & Clinical Development

24 March 2020

published in

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volume

8

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24 March 2020

page(s)

152-165

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describes a project that uses

24 March 2020

ImageJ

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11 June 2022

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inferred from PubMed Central ID database lookup

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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24 June 2018

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Late-onset MNGIE without peripheral neuropathy due to incomplete loss of thymidine phosphorylase activity

13 August 2018

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Pre- and post-dialysis quantitative dosage of thymidine in urine and plasma of a MNGIE patient by using HPLC-ESI-MS/MS.

13 August 2018

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Late-onset MNGIE due to partial loss of thymidine phosphorylase activity.

13 August 2018

1 reference

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Increased blood-brain barrier permeability with thymidine phosphorylase deficiency

13 August 2018

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13 August 2018

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2 November 2018

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12 December 2020

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12 December 2020

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12 December 2020

High mitochondrial DNA copy number has detrimental effects in mice

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Identifiers

DOI

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:29687034%20AND%20SRC:MED&resulttype=core&format=json

24 March 2020

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24 March 2020

1 reference