Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA, Treatment of adrenoleukodystrophy

Case Medical Research ◽

10.31525/cmr-cc49d1 ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Haematopoietic Stem Cells ◽

Orphan Designation

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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene, Treatment of beta thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-de550e ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Globin Gene ◽

Haematopoietic Stem Cells ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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Orphan designation: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA, Treatment of adrenoleukodystrophy

Case Medical Research ◽

10.31525/cmr-cc982f ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Haematopoietic Stem Cells ◽

Orphan Designation

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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene for the treatment of sickle cell disease, The treatment of sickle cell disease

Case Medical Research ◽

10.31525/cmr-26af3b3 ◽

2020 ◽

Author(s):

Keyword(s):

Sickle Cell Disease ◽

Sickle Cell ◽

Lentiviral Vector ◽

Globin Gene ◽

Cell Disease ◽

Haematopoietic Stem Cells ◽

Orphan Designation

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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene, Treatment of beta thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-de7554 ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Globin Gene ◽

Haematopoietic Stem Cells ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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EXPRESSION OF HUMAN FACTOR IX GENE IN MURINE PLASMA THROUGH LENTIVIRAL VECTOR-INFECTED HAEMATOPOIETIC STEM CELLS

Clinical and Experimental Pharmacology and Physiology ◽

10.1111/j.1440-1681.2006.04511.x ◽

2006 ◽

Vol 33 (12) ◽

pp. 1196-1201 ◽

Author(s):

Haoming Chen ◽

Hengmei Yao ◽

Lu Huang ◽

Qi Shen ◽

William Jia ◽

...

Keyword(s):

Human Factor ◽

Lentiviral Vector ◽

Factor Ix ◽

Haematopoietic Stem Cells ◽

Human Factor Ix

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F2A sequence linking MGMTP140K and MDR1 in a bicistronic lentiviral vector enables efficient chemoprotection of haematopoietic stem cells

Cancer Gene Therapy ◽

10.1038/cgt.2012.67 ◽

2012 ◽

Vol 19 (11) ◽

pp. 802-810 ◽

Author(s):

P Maier ◽

D Heckmann ◽

I Spier ◽

S Laufs ◽

M Zucknick ◽

...

Keyword(s):

Lentiviral Vector ◽

Haematopoietic Stem Cells

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Orphan designation: Autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-277ad49 ◽

2020 ◽

Author(s):

Keyword(s):

Haematopoietic Stem Cells ◽

Enhancer Region ◽

Orphan Designation ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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Engraftment of T-cell-depleted allogeneic haematopoietic stem cells using a reduced intensity conditioning regimen

British Journal of Haematology ◽

10.1046/j.1365-2141.2000.02454.x ◽

2000 ◽

Vol 111 (3) ◽

pp. 797-800 ◽

Author(s):

C. Craddock ◽

P. Bardy ◽

S. Kreiter ◽

R. Johnston ◽

J. Apperley ◽

...

Keyword(s):

T Cell ◽

Conditioning Regimen ◽

Reduced Intensity Conditioning ◽

Haematopoietic Stem Cells ◽

Reduced Intensity Conditioning Regimen ◽

Reduced Intensity

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Faculty Opinions recommendation of Deficiencies in DNA damage repair limit the function of haematopoietic stem cells with age.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.1087486.540536 ◽

2007 ◽

Author(s):

Lucio Comai

Keyword(s):

Dna Damage Repair ◽

Haematopoietic Stem Cells ◽

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Faculty Opinions recommendation of DNA repair is limiting for haematopoietic stem cells during ageing.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.1087465.550780 ◽

2007 ◽

Author(s):

Dale Ramsden

Keyword(s):

Haematopoietic Stem Cells

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