(Q35651113)

9 August 2017

title

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells (English)

1 reference

9 August 2017

Zulema Romero

1

1 reference

9 August 2017

Beatriz Campo-Fernandez

2

1 reference

9 August 2017

Jennifer Wherley

3

1 reference

9 August 2017

Michael L Kaufman

4

1 reference

9 August 2017

Fabrizia Urbinati

5

1 reference

9 August 2017

Aaron R Cooper

6

1 reference

9 August 2017

Megan D Hoban

7

1 reference

9 August 2017

Kismet M Baldwin

8

1 reference

9 August 2017

Dianne Lumaquin

9

1 reference

9 August 2017

Xiaoyan Wang

10

1 reference

9 August 2017

Shantha Senadheera

11

1 reference

9 August 2017

Roger P Hollis

12

1 reference

9 August 2017

Donald B Kohn

13

1 reference

9 August 2017

publication date

22 April 2015

1 reference

Molecular Therapy. Methods & Clinical Development

9 August 2017

published in

1 reference

9 August 2017

volume

2

1 reference

9 August 2017

page(s)

15012

1 reference

β-globin gene transfer to human bone marrow for sickle cell disease.

9 August 2017

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

High-throughput droplet digital PCR system for absolute quantitation of DNA copy number

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Highly efficient large-scale lentiviral vector concentration by tandem tangential flow filtration

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

The use of chromatin insulators to improve the expression and safety of integrating gene transfer vectors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Mutation of a barrier insulator in the human ankyrin-1 gene is associated with hereditary spherocytosis

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Blood and marrow transplantation for sickle cell disease: overcoming barriers to success

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Correction of murine sickle cell disease using gamma-globin lentiviral vectors to mediate high-level expression of fetal hemoglobin

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Combinatorial incorporation of enhancer-blocking components of the chicken beta-globin 5'HS4 and human T-cell receptor alpha/delta BEAD-1 insulators in self-inactivating retroviral vectors reduces their genotoxic potential

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

USF1 recruits histone modification complexes and is critical for maintenance of a chromatin barrier

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Pathophysiology and therapy for haemoglobinopathies. Part I: sickle cell disease

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Silencing and variegation of gammaretrovirus and lentivirus vectors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

12 July 2018

Locus control regions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Correction of sickle cell disease in transgenic mouse models by gene therapy

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

The beta-globin locus control region versus gene therapy vectors: a struggle for expression.

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Inefficient processing impairs release of RNA from the site of transcription

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

The dynamics of globin gene expression and gene therapy vectors

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Efficient 3'-end formation of human beta-globin mRNA in vivo requires sequences within the last intron but occurs independently of the splicing reaction

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

A dominant chromatin-opening activity in 5' hypersensitive site 3 of the human beta-globin locus control region

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Position independence and proper developmental control of gamma-globin gene expression require both a 5' locus control region and a downstream sequence element

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Ex vivo generation of human red blood cells: a new advance in stem cell engineering

12 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Improved human beta-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element

25 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Successful correction of the human beta-thalassemia major phenotype using a lentiviral vector

25 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Correction of a mouse model of sickle cell disease: lentiviral/antisickling beta-globin gene transduction of unmobilized, purified hematopoietic stem cells

25 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4445009

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

25 September 2018

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FMTM.2015.12

inferred from DOI database lookup

7 January 2021

based on heuristic

Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FMTM.2015.12

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/MTM.2015.12

1 reference

9 August 2017

1 reference

9 August 2017

1 reference