(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

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

5 March 2020

0 references

1 reference

4

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

Ivano Di Meo

5

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

Jordi Barquinero

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

Alberto Auricchio

7

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

Ramon Martí

11

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

10

Massimo Zeviani

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

2

Carlo Viscomi

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

author name string

Javier Torres-Torronteras

1

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

Raquel Cabrera-Pérez

3

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

Giuseppe Pizzorno

8

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

Michio Hirano

9

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

language of work or name

1 reference

22 January 2014

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

number of pages

7

1 reference

based on heuristic

inferred from page(s)

published in

Molecular Therapy

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

volume

22

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

issue

5

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

page(s)

901-907

1 reference

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

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

5 March 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Clinical and genetic spectrum of mitochondrial neurogastrointestinal encephalomyopathy

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Adeno-associated virus integration: virus versus vector

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Allogeneic stem cell transplantation corrects biochemical derangements in MNGIE

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Mammalian nucleoside transporters

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Altered thymidine metabolism due to defects of thymidine phosphorylase

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Thymidine phosphorylase gene mutations in MNGIE, a human mitochondrial disorder

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

11 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Treatment of mitochondrial neurogastrointestinal encephalomyopathy with dialysis

25 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

No tumour-initiating risk associated with scAAV transduction in newborn rat liver.

17 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Development and implementation of standardized respiratory chain spectrophotometric assays for clinical diagnosis

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Carrier erythrocyte entrapped thymidine phosphorylase therapy for MNGIE.

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Purification of a crude mitochondrial fraction by density-gradient centrifugation

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Elevated plasma deoxyuridine in patients with thymidine phosphorylase deficiency

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4015233

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