Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
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Advances of gene therapy for primary immunodeficienciesClinical development of gene therapy: results and lessons from recent successesGene therapy: progress and predictionsGene therapy on the moveRetroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectivesAdvances in salivary gland gene therapy - oral and systemic implicationsHuman RAG mutations: biochemistry and clinical implicationsDevelopment of Hematopoietic Stem Cell Based Gene Therapy for HIV-1 Infection: Considerations for Proof of Concept Studies and Translation to Standard Medical PracticeGene therapy for PIDs: progress, pitfalls and prospectsAssessment of Immature Platelet Fraction in the Diagnosis of Wiskott-Aldrich Syndrome.Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.β-globin gene transfer to human bone marrow for sickle cell disease.Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Single Cell-Based Vector Tracing in Patients with ADA-SCID Treated with Stem Cell Gene Therapy.HSV-sr39TK positron emission tomography and suicide gene elimination of human hematopoietic stem cells and their progeny in humanized mice.Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.Gene therapy for inherited immunodeficiency.Mouse transplant models for evaluating the oncogenic risk of a self-inactivating XSCID lentiviral vectorA modified γ-retrovirus vector for X-linked severe combined immunodeficiency.Cell cycle status of CD34(+) hemopoietic stem cells determines lentiviral integration in actively transcribed and development-related genes.Clinical applications of gene therapy for primary immunodeficiencies.Effects of vector backbone and pseudotype on lentiviral vector-mediated gene transfer: studies in infant ADA-deficient mice and rhesus monkeys.History and current status of newborn screening for severe combined immunodeficiency.Large-scale production of lentiviral vector in a closed system hollow fiber bioreactor.Prevention of Lethal Murine Hypophosphatasia by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow CellsB-cell development and functions and therapeutic options in adenosine deaminase-deficient patients.An assessment of the effects of ectopic gp91phox expression in XCGD iPSC-derived neutrophils.Gene Therapy: The View from NCATS.Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.Cost-Effectiveness/Cost-Benefit Analysis of Newborn Screening for Severe Combined Immune Deficiency in Washington StateHuman Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington's Disease Mouse Models.A Low Protein Binding Cationic Poly(2-oxazoline) as Non-Viral VectorIn Vivo Tracking of Human Hematopoiesis Reveals Patterns of Clonal Dynamics during Early and Steady-State Reconstitution PhasesDevelopment of gene therapy for blood disorders: an update.A comparison of foamy and lentiviral vector genotoxicity in SCID-repopulating cells shows foamy vectors are less prone to clonal dominance.Neutropenia in primary immunodeficiencyGenetic Tagging During Human Mesoderm Differentiation Reveals Tripotent Lateral Plate Mesodermal Progenitors
P2860
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P2860
Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
description
2012 nî lūn-bûn
@nan
2012年の論文
@ja
2012年学术文章
@wuu
2012年学术文章
@zh-cn
2012年学术文章
@zh-hans
2012年学术文章
@zh-my
2012年学术文章
@zh-sg
2012年學術文章
@yue
2012年學術文章
@zh
2012年學術文章
@zh-hant
name
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@ast
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@en
type
label
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@ast
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@en
prefLabel
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@ast
Gene therapy for adenosine dea ...... l vectors and treatment plans.
@en
P2093
P2860
P50
P1433
P1476
Gene therapy for adenosine dea ...... al vectors and treatment plans
@en
P2093
Alan S Wayne
Arumugam Balamurugan
Barbara C Engel
Carla M Davis
Celine Hanson
Christopher Choi
David Gjertson
Denise Carbonaro
Elizabeth Garabedian
P2860
P304
P356
10.1182/BLOOD-2012-02-400937
P407
P577
2012-09-11T00:00:00Z