A universal system to select gene-modified hepatocytes in vivo (Q28468539)

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

author name string

Sean Nygaard

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

Adi Barzel

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

Annelise Haft

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

Angela Major

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

Milton Finegold

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

Mark A Kay

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

Science Translational Medicine

8 June 2020

language of work or name

English

0 references

publication date

8 June 2016

0 references

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

volume

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

issue

342

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

page(s)

342ra79

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

In vivo genome editing restores haemostasis in a mouse model of haemophilia

8 June 2020

cites work

1 reference

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Mechanistic inferences from the crystal structure of fumarylacetoacetate hydrolase with a bound phosphorus-based inhibitor

19 March 2017

1 reference

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Complete rescue of lethal albino c14CoS mice by null mutation of 4-hydroxyphenylpyruvate dioxygenase and induction of apoptosis of hepatocytes in these mice by in vivo retrieval of the tyrosine catabolic pathway

19 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

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

19 March 2017

1 reference

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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

7 April 2017

1 reference

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Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

29 September 2017

1 reference

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Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

29 September 2017

1 reference

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

29 September 2017

1 reference

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Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients

29 September 2017

1 reference

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Genetic basis of drug-induced liver injury: present and future

29 September 2017

1 reference

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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

29 September 2017

1 reference

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An optimized microRNA backbone for effective single-copy RNAi

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Effect of nitisinone (NTBC) treatment on the clinical course of hepatorenal tyrosinemia in Québec

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Induction of hepatocellular carcinoma by in vivo gene targeting

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Metabolic activation in drug-induced liver injury

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Spontaneous hepatic repopulation in transgenic mice expressing mutant human α1-antitrypsin by wild-type donor hepatocytes

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

TBI during BM and SCT: review of the past, discussion of the present and consideration of future directions

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Immune responses to AAV in clinical trials

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Slow-onset inhibition of fumarylacetoacetate hydrolase by phosphinate mimics of the tetrahedral intermediate: kinetics, crystal structure and pharmacokinetics.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

29 September 2017

1 reference

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Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6.

29 September 2017

1 reference

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Kinetics of liver repopulation after bone marrow transplantation.

29 September 2017

1 reference

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Nontransplant treatment of tyrosinemia

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

In vivo suppressor mutations correct a murine model of hereditary tyrosinemia type I.

29 September 2017

1 reference

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Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Self-induced correction of the genetic defect in tyrosinemia type I

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

AAV vector integration sites in mouse hepatocellular carcinoma.

2 June 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Gene targeting in vivo by adeno-associated virus vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

In vivo selection and chemoprotection after drug resistance gene therapy in a nonmyeloablative allogeneic transplantation setting in dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Adeno-associated virus finds its disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Pharmacological correction of neonatal lethal hepatic dysfunction in a murine model of hereditary tyrosinaemia type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAD8166

Cyclin B-dependent kinase and caspase-1 activation precedes mitochondrial dysfunction in fumarylacetoacetate-induced apoptosis.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5242329

Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in Long-Evans cinnamon rats

27 November 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27280686

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Therapeutic liver repopulation in a mouse model of hypercholesterolemia

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27280686

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1126/SCITRANSLMED.AAD8166

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27280686%20AND%20SRC:MED&resulttype=core&format=json

8 June 2020

1 reference