(Q34587615)

3 August 2017

1 reference

title

Globin gene transfer for the treatment of severe hemoglobinopathies: a paradigm for stem cell-based gene therapy (English)

1 reference

3 August 2017

0 references

Michel Sadelain

1

1 reference

3 August 2017

language of work or name

1 reference

1 March 2002

1 reference

3 August 2017

published in

Journal of Gene Medicine

1 reference

3 August 2017

volume

4

1 reference

3 August 2017

issue

2

1 reference

3 August 2017

page(s)

113-121

1 reference

The molecular basis of hemoglobin disease

3 August 2017

cites work

1 reference

Regulation of Globin Gene Expression in Erythroid Cells

21 January 2018

1 reference

Regulation of hemoglobin synthesis during the development of the red cell (first of three parts).

21 January 2018

1 reference

Quantitative measurement of the erythrocytic and granulocytic cells of the marrow and blood

21 January 2018

1 reference

Thalassemia--a global public health problem

21 January 2018

1 reference

Therapeutic approaches to hemoglobin switching in treatment of hemoglobinopathies

21 January 2018

1 reference

21 January 2018

The beta-thalassemias

1 reference

New trends in the treatment of beta-thalassemia

21 January 2018

1 reference

Sickle cell anemia, a molecular disease

21 January 2018

1 reference

Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia

21 January 2018

1 reference

Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia

21 January 2018

1 reference

Genetic treatment of the haemoglobinopathies: recombinations and new combinations

21 January 2018

1 reference

Gene therapy for the hemoglobin disorders: past, present, and future

21 January 2018

1 reference

The "beta-like-globin" gene domain in human erythroid cells

21 January 2018

1 reference

Evidence for a locus activation region: the formation of developmentally stable hypersensitive sites in globin-expressing hybrids

21 January 2018

1 reference

Position-independent, high-level expression of the human beta-globin gene in transgenic mice

21 January 2018

1 reference

Locus control regions: coming of age at a decade plus

21 January 2018

1 reference

Locus control regions of mammalian beta-globin gene clusters: combining phylogenetic analyses and experimental results to gain functional insights

21 January 2018

1 reference

Human beta-globin locus control region: analysis of the 5' DNase I hypersensitive site HS 2 in transgenic mice

21 January 2018

1 reference

Hypersensitive site 4 of the human beta globin locus control region

21 January 2018

1 reference

Dissection of the enhancer activity of beta-globin 5' DNase I-hypersensitive site 2 in transgenic mice

21 January 2018

1 reference

Regulation of beta-globin gene expression: straightening out the locus.

21 January 2018

1 reference

Activation by locus control regions?

21 January 2018

1 reference

Looping versus linking: toward a model for long-distance gene activation

21 January 2018

1 reference

Locus control regions, chromatin activation and transcription

21 January 2018

1 reference

Targeted deletion of 5'HS1 and 5'HS4 of the beta-globin locus control region reveals additive activity of the DNaseI hypersensitive sites

21 January 2018

1 reference

Regulated expression of a complete human beta-globin gene encoded by a transmissible retrovirus vector

21 January 2018

1 reference

Retroviral-mediated transfer of genomic globin genes leads to regulated production of RNA and protein

21 January 2018

1 reference

Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retrovirus-transduced stem cells

21 January 2018

1 reference

Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells

21 January 2018

1 reference

A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells

21 January 2018

1 reference

Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene

21 January 2018

1 reference

A 36-base-pair core sequence of locus control region enhances retrovirally transferred human beta-globin gene expression

21 January 2018

1 reference

High-level beta-globin expression after retroviral transfer of locus activation region-containing human beta-globin gene derivatives into murine erythroleukemia cells

21 January 2018

1 reference

Evaluation of beta-globin gene therapy constructs in single copy transgenic mice.

21 January 2018

1 reference

A promising genetic approach to the treatment of beta-thalassemia

21 January 2018

1 reference

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

21 January 2018

1 reference

The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA

21 January 2018

1 reference

HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells

21 January 2018

1 reference

Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

21 January 2018

1 reference

Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors

21 January 2018

1 reference

Issues in the manufacture and transplantation of genetically modified hematopoietic stem cells

21 January 2018

1 reference

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

21 January 2018

1 reference

Long-term expression of gamma-globin mRNA in mouse erythrocytes from retrovirus vectors containing the human gamma-globin gene fused to the ankyrin-1 promoter

21 January 2018

1 reference

High-level erythroid-specific gene expression in primary human and murine hematopoietic cells with self-inactivating lentiviral vectors

21 January 2018

1 reference

Boundary and insulator elements in chromosomes

21 January 2018

1 reference

A 5' element of the chicken beta-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila

21 January 2018

1 reference

The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites.

21 January 2018

1 reference

A chromatin insulator protects retrovirus vectors from chromosomal position effects

21 January 2018

1 reference

Gene transfer into hematopoietic stem cells: long-term maintenance of in vitro activated progenitors without marrow ablation

21 January 2018

1 reference

Special proliferative sites are not needed for seeding and proliferation of transfused bone marrow cells in normal syngeneic mice

21 January 2018

1 reference

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

1 reference

Selective erythroid replacement in murine beta-thalassemia using fetal hematopoietic stem cells

21 January 2018

1 reference

Inactivation of the human beta-globin gene by targeted insertion into the beta-globin locus control region

21 January 2018

1 reference

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

21 January 2018

1 reference

Toward gene therapy for disorders of globin synthesis

21 January 2018

1 reference

A simple disease with no cure

21 January 2018

1 reference

The structure and evolution of the human beta-globin gene family

21 January 2018

1 reference

Bone marrow transplantation in thalassemia. The experience of Pesaro

21 January 2018

1 reference

Bone marrow transplantation for homozygous beta-thalassemia. The Memorial Sloan-Kettering Cancer Center experience

21 January 2018

1 reference

Bone marrow transplantation for sickle cell anemia

21 January 2018

1 reference

A 3' enhancer contributes to the stage-specific expression of the human beta-globin gene

21 January 2018

1 reference

A foreign beta-globin gene in transgenic mice: integration at abnormal chromosomal positions and expression in inappropriate tissues

21 January 2018

1 reference

The beta-globin dominant control region activates homologous and heterologous promoters in a tissue-specific manner

21 January 2018

1 reference

Do LCRs open chromatin domains?

21 January 2018

1 reference

Successful treatment of murine beta-thalassemia intermedia by transfer of the human beta-globin gene

21 January 2018

1 reference

Implantation of normal bloodforming tissue in genetically anemic mice, without x-irradiation of host

21 January 2018

1 reference

21 January 2018

Identifiers

DOI

10.1002/JGM.266

1 reference

3 August 2017

1 reference