(Q35829187)

10 August 2017

Fetal gene therapy of alpha-thalassemia in a mouse model (English)

1 reference

10 August 2017

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Xiao-Dong Han

1

1 reference

10 August 2017

Chin Lin

2

1 reference

10 August 2017

Judy Chang

3

1 reference

10 August 2017

Michel Sadelain

4

1 reference

10 August 2017

Y W Kan

5

1 reference

10 August 2017

language of work or name

English

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publication date

11 May 2007

1 reference

Proceedings of the National Academy of Sciences of the United States of America

10 August 2017

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10 August 2017

104

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10 August 2017

21

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10 August 2017

9007-9011

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Successful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector

10 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Successful mismatched sibling cord blood transplant in Hb Bart's disease

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Temporally regulated expression patterns following in utero adenovirus-mediated gene transfer

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors.

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

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

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Origin and fate of the central macrophages of erythroblastic islands in the fetal and neonatal mouse liver

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Human alpha-globin genes demonstrate autonomous developmental regulation in transgenic mice

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Prenatal diagnosis of alpha-thalassemia. Clinical application of molecular hybridization

11 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

Homozygous alpha-thalassemia treated with intrauterine transfusions and postnatal hematopoietic stem cell transplantation

11 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

Treatment of hemoglobin Bart's hydrops with bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

Widespread gene transduction to the central nervous system by adenovirus in utero: implication for prenatal gene therapy to brain involvement of lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0702457104

Inactivation of mouse alpha-globin gene by homologous recombination: mouse model of hemoglobin H disease

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=1885618

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

24 September 2018

1 reference

12 December 2020

Ontogeny of the Haemopoietic System: Yolk Sac Origin of In Vivo and In Vitro Colony Forming Cells in the Developing Mouse Embryo

1 reference

12 December 2020

Survival of a hydropic infant with homozygous α-thalassemia-1

1 reference

12 December 2020

Normal long-term survival with alpha-thalassemia

1 reference

12 December 2020

Identifiers

DOI

10.1073/PNAS.0702457104

1 reference

10 August 2017

0 references

1 reference

10 August 2017

PubMed publication ID

17496141

1 reference