Correction of sickle cell disease in transgenic mouse models by gene therapy.
about
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaCurrent and future alternative therapies for beta-thalassemia majorPluripotent stem cells in research and treatment of hemoglobinopathiesState of the art: stem cells in equine regenerative medicineAnemia: progress in molecular mechanisms and therapiesBeta-globin LCR and intron elements cooperate and direct spatial reorganization for gene therapyAnimal models of β-hemoglobinopathies: utility and limitationsThe potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesAdvances in sickle cell therapies in the hydroxyurea eraHematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integrationTherapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patientsMacrophage-mediated GDNF delivery protects against dopaminergic neurodegeneration: a therapeutic strategy for Parkinson's disease.Development of B-lineage predominant lentiviral vectors for use in genetic therapies for B cell disordersIdentification of synthetic endothelial cell-specific promoters by use of a high-throughput screen.Combinatorial evaluation of cations, pH-sensitive and hydrophobic moieties for polymeric vector designGene specificity of suppression of transgene-mediated insertional transcriptional activation by the chicken HS4 insulator.β-globin gene transfer to human bone marrow for sickle cell disease.Differences of globin transgene expression in stably transfected cell lines and transgenic mice.Effects of human gamma-globin in murine beta-thalassaemia.Transient in vivo beta-globin production after lentiviral gene transfer to hematopoietic stem cells in the nonhuman primateEx vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsinFree heme and the polymerization of sickle cell hemoglobinGene therapy for hemoglobinopathies: the state of the field and the future.Permanent and panerythroid correction of murine beta thalassemia by multiple lentiviral integration in hematopoietic stem cells.Lentiviral vectors, two decades later.Globin gene transfer for the treatment of severe hemoglobinopathies: a paradigm for stem cell-based gene therapy.Determinants of specific RNA interference-mediated silencing of human beta-globin alleles differing by a single nucleotide polymorphism.Developing treatment for sickle cell disease.High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivoTherapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer.In utero therapy for congenital disorders using amniotic fluid stem cells.Spontaneous circulation of myeloid-lymphoid-initiating cells and SCID-repopulating cells in sickle cell crisis.Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion.Therapeutic challenges in childhood sickle cell disease. Part 1: current and future treatment options.Distribution of lentiviral vector integration sites in mice following therapeutic gene transfer to treat β-thalassemia.Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now.Current status of gene delivery and gene therapy in lacrimal gland using viral vectors.Lentiviral transgene vectorsThe human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells.Targeted gene modification of hematopoietic progenitor cells in mice following systemic administration of a PNA-peptide conjugate
P2860
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P2860
Correction of sickle cell disease in transgenic mouse models by gene therapy.
description
2001 nî lūn-bûn
@nan
2001 թուականի Դեկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի դեկտեմբերին հրատարակված գիտական հոդված
@hy
2001年の論文
@ja
2001年論文
@yue
2001年論文
@zh-hant
2001年論文
@zh-hk
2001年論文
@zh-mo
2001年論文
@zh-tw
2001年论文
@wuu
name
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@ast
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@en
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@nl
type
label
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@ast
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@en
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@nl
prefLabel
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@ast
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@en
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@nl
P2093
P356
P1433
P1476
Correction of sickle cell disease in transgenic mouse models by gene therapy.
@en
P2093
Acharya SA
Bouhassira EE
Humphries RK
Leboulch P
P304
P356
10.1126/SCIENCE.1065806
P407
P577
2001-12-01T00:00:00Z