(Q34621690)

English

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I

scientific article

In more languages

Statements

scholarly article

1 reference

Europe PubMed Central

3 August 2017

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I (English)

1 reference

Europe PubMed Central

3 August 2017

0 references

mucopolysaccharidosis I

1 reference

based on heuristic

inferred from title

9

1 reference

Europe PubMed Central

3 August 2017

7

object named as

Luigi Naldini

0 references

10

object named as

Bruce R Blazar

1 reference

Europe PubMed Central

3 August 2017

Gordon J. Freeman

8

object named as

Gordon Freeman

1 reference

Europe PubMed Central

3 August 2017

author name string

Mark J Osborn

1

1 reference

Europe PubMed Central

3 August 2017

Ron T McElmurry

2

1 reference

Europe PubMed Central

3 August 2017

Christopher J Lees

3

1 reference

Europe PubMed Central

3 August 2017

Anthony P DeFeo

4

1 reference

Europe PubMed Central

3 August 2017

Zhi-Ying Chen

5

1 reference

Europe PubMed Central

3 August 2017

Mark A Kay

6

1 reference

Europe PubMed Central

3 August 2017

language of work or name

1 reference

based on heuristic

inferred from title

publication date

16 November 2010

1 reference

Europe PubMed Central

3 August 2017

Molecular Therapy

1 reference

Europe PubMed Central

3 August 2017

19

1 reference

Europe PubMed Central

3 August 2017

450-460

1 reference

Europe PubMed Central

3 August 2017

3

1 reference

Europe PubMed Central

3 August 2017

Gender-related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

alpha-L-iduronidase therapy for mucopolysaccharidosis type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Effect of tissue-specific promoters and microRNA recognition elements on stability of transgene expression after hydrodynamic naked plasmid DNA delivery

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Image-guided, lobe-specific hydrodynamic gene delivery to swine liver

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Stable knockdown of microRNA in vivo by lentiviral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Lentiviral-vector-mediated expression of murine IL-1 receptor antagonist or IL-10 reduces the severity of endotoxin-induced uveitis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Transient blockade of the inducible costimulator pathway generates long-term tolerance to factor VIII after nonviral gene transfer into hemophilia A mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

The cHS4 chromatin insulator reduces gammaretroviral vector silencing by epigenetic modifications of integrated provirus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Identification and analysis of functional elements in 1% of the human genome by the ENCODE pilot project

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Reducing the immunostimulatory activity of CpG-containing plasmid DNA vectors for non-viral gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Increased maintenance and persistence of transgenes by excision of expression cassettes from plasmid sequences in vivo.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

The phenotypic characters of the stem cells in hepatocytes during liver regeneration: the expression of the Bcrp1/Abcg2 membrane transporter and the Hoechst 33342 export

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Transcription start regions in the human genome are favored targets for MLV integration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

ICOS is critical for CD40-mediated antibody class switching

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

7 July 2018

Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Effect of neonatal administration of a retroviral vector expressing alpha-L-iduronidase upon lysosomal storage in brain and other organs in mucopolysaccharidosis I mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Dynamic gene expression after systemic delivery of plasmid DNA as determined by in vivo bioluminescence imaging.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Hydrodynamic liver gene transfer mechanism involves transient sinusoidal blood stasis and massive hepatocyte endocytic vesicles.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Targeting of inducible costimulator (ICOS) expressed on alloreactive T cells down-regulates graft-versus-host disease (GVHD) and facilitates engraftment of allogeneic bone marrow (BM).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Induction of tolerance to human factor VIII in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3048178

26 October 2018

The presenting features of mucopolysaccharidosis type IH (Hurler syndrome)

1 reference

https://pubmed.ncbi.nlm.nih.gov/21081900

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Ligand binding specificities of the two mannose 6-phosphate receptors

1 reference

https://pubmed.ncbi.nlm.nih.gov/21081900

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

1 reference

https://api.crossref.org/works/10.1038%2FMT.2010.249

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1038/MT.2010.249

1 reference

Europe PubMed Central

3 August 2017

1 reference

Europe PubMed Central

3 August 2017

1 reference

Europe PubMed Central

3 August 2017

ResearchGate publication ID

0 references

Sitelinks

Wikipedia(0 entries)

Wikibooks(0 entries)

Wikinews(0 entries)

Wikiquote(0 entries)

Wikisource(0 entries)

Wikiversity(0 entries)

Wikivoyage(0 entries)

Wiktionary(0 entries)

Multilingual sites(0 entries)

Retrieved from "https://www.wikidata.org/w/index.php?title=Q34621690&oldid=2134833272"