Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
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Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectivesGene 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 2017: Progress and Future Directions.Alterations in the brain adenosine metabolism cause behavioral and neurological impairment in ADA-deficient mice and patients.Gene therapy for primary immune deficiencies: a Canadian perspective.Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency.Precision medicine, genomics and drug discovery.Gene Therapy with Hematopoietic Stem Cells: The Diseased Bone Marrow's Point of View.How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID).New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.Evolving Gene Therapy in Primary Immunodeficiency.Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.HIV-1-mediated insertional activation of STAT5B and BACH2 trigger viral reservoir in T regulatory cells.Stimulus-responsive viral vectors for controlled delivery of therapeutics.Biological and functional characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency.Haematopoietic stem cell transplantation for primary immunodeficiency syndromes: A 5-year single-centre experience.Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies.Multiparametric Whole Blood Dissection: A one-shot comprehensive picture of the human hematopoietic system.Long term outcomes of severe combined immunodeficiency: therapy implications.Immobilized hematopoietic growth factors onto magnetic particles offer a scalable strategy for cell therapy manufacturing in suspension cultures.First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature.Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID.From clinical observations and molecular dissection to novel therapeutic strategies for primary immunodeficiency disorders.Adenosine deaminase deficiency: a review.Cholesteryl oleate-loaded cationic solid lipid nanoparticles as carriers for efficient gene-silencing therapy.
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P2860
Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 29 April 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Update on the safety and effic ...... denosine deaminase deficiency.
@en
Update on the safety and effic ...... denosine deaminase deficiency.
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type
label
Update on the safety and effic ...... denosine deaminase deficiency.
@en
Update on the safety and effic ...... denosine deaminase deficiency.
@nl
prefLabel
Update on the safety and effic ...... denosine deaminase deficiency.
@en
Update on the safety and effic ...... denosine deaminase deficiency.
@nl
P2093
P2860
P1433
P1476
Update on the safety and effic ...... adenosine deaminase deficiency
@en
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Antonella Tabucchi
Erika De Boever
Eyal Grunebaum
Fabio Ciceri
Federica Barzaghi
Filippo Carlucci
Francesca Dionisio
Ilhan Tezcan
Immacolata Brigida
Jennifer M Puck
P2860
P356
10.1182/BLOOD-2016-01-688226
P407
P577
2016-04-29T00:00:00Z