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

Case Medical Research ◽

10.31525/cmr-12043ed ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Globin Gene ◽

Beta Globin ◽

Orphan Designation ◽

Beta Globin Gene ◽

Beta Thalassaemia ◽

Gene Treatment ◽

Thalassaemia Intermedia

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Orphan designation: 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridine-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-1e36742 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-ea7c31 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Covalently Linked ◽

Thalassaemia Intermedia ◽

Sirna Oligonucleotide

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Orphan designation: human apotransferrin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-bb17bc ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

Download Full-text

Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-ea2d19 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Covalently Linked ◽

Thalassaemia Intermedia ◽

Sirna Oligonucleotide

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Orphan designation: human apotransferrin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-bc10f9 ◽

2019 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

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-cc49d1 ◽

2019 ◽

Author(s):

Keyword(s):

Lentiviral Vector ◽

Haematopoietic Stem Cells ◽

Orphan Designation

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Orphan designation: Bitopertin, Treatment of beta-thalassaemia intermedia and major

Case Medical Research ◽

10.31525/cmr-3996c1 ◽

2018 ◽

Author(s):

Keyword(s):

Orphan Designation ◽

Beta Thalassaemia ◽

Thalassaemia Intermedia

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