In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia.
about
Genetic modification of somatic stem cells. The progress, problems and prospects of a new therapeutic technologyTransfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaCurrent and future alternative therapies for beta-thalassemia majorThe GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgeneAnimal models of β-hemoglobinopathies: utility and limitationsTherapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patientsIn vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle deliveryβ-globin gene transfer to human bone marrow for sickle cell disease.Future alternative therapies for β-thalassemia.Role of the GATA-1/FOG-1/NuRD pathway in the expression of human beta-like globin genes.Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease.Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells.Genetic control of wayward pluripotent stem cells and their progeny after transplantation.Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugateThe new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells.Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcriptsCorrection of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.The β-globin locus control region in combination with the EF1α short promoter allows enhanced lentiviral vector-mediated erythroid gene expression with conserved multilineage activityGene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.Gene therapy for hemoglobinopathies: progress and challenges.Amelioration of murine beta-thalassemia through drug selection of hematopoietic stem cells transduced with a lentiviral vector encoding both gamma-globin and the MGMT drug-resistance gene.Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.Gene therapy for β-thalassaemia: the continuing challenge.Gene therapy in thalassemia and hemoglobinopathies.Alternative options for DNA-based experimental therapy of β-thalassemia.Recent advances in gene therapy for thalassemia.Recent trends in the gene therapy of β-thalassemia.Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.Measurement of lentiviral vector titre and copy number by cross-species duplex quantitative PCR.Gene Therapy for β-Hemoglobinopathies.Comparative analysis of FV vectors with human α- or β-globin gene regulatory elements for the correction of β-thalassemia.The Ongoing Challenge of Hematopoietic Stem Cell-Based Gene Therapy for β-Thalassemia.Recent advances in β-thalassemiasGenome editing and stem cell therapy pave the path for new treatment of sickle-cell disease.Split chimerism between nucleated and red blood cells after bone marrow transplantation for haemoglobinopathies.Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies.Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia.
P2860
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P2860
In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
2008年论文
@zh
2008年论文
@zh-cn
name
In vivo selection of genetical ...... orrection of beta-thalassemia.
@ast
In vivo selection of genetical ...... orrection of beta-thalassemia.
@en
type
label
In vivo selection of genetical ...... orrection of beta-thalassemia.
@ast
In vivo selection of genetical ...... orrection of beta-thalassemia.
@en
prefLabel
In vivo selection of genetical ...... orrection of beta-thalassemia.
@ast
In vivo selection of genetical ...... orrection of beta-thalassemia.
@en
P2093
P2860
P356
P1476
In vivo selection of genetical ...... orrection of beta-thalassemia.
@en
P2093
Annarita Miccio
Cheok-Man Chow
Claudia Rossi
Francesca Sanvito
Francesco Lotti
Giuliana Ferrari
Maurilio Ponzoni
Rossano Cesari
Samantha J E Routledge
P2860
P304
10547-10552
P356
10.1073/PNAS.0711666105
P407
P577
2008-07-23T00:00:00Z