Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice.
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A transposon and transposase system for human applicationThe Sleeping Beauty transposon system: a non-viral vector for gene therapyAnimal models of hemophiliaGene therapy for hemophiliaTenfibgen ligand nanoencapsulation delivers bi-functional anti-CK2 RNAi oligomer to key sites for prostate cancer targeting using human xenograft tumors in miceNovel therapeutic approaches for various cancer types using a modified sleeping beauty-based gene delivery systemGene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons.CK2 modulation of NF-kappaB, TP53, and the malignant phenotype in head and neck cancer by anti-CK2 oligonucleotides in vitro or in vivo via sub-50-nm nanocapsules.Advancements in gene transfer-based therapy for hemophilia A.Mechanism and efficacy of sub-50-nm tenfibgen nanocapsules for cancer cell-directed delivery of anti-CK2 RNAi to primary and metastatic squamous cell carcinoma.Emergence of protein kinase CK2 as a key target in cancer therapyTranslating Sleeping Beauty transposition into cellular therapies: victories and challenges.Targeting FVIII expression to endothelial cells regenerates a releasable pool of FVIII and restores hemostasis in a mouse model of hemophilia A.Inhibition of angiogenesis and suppression of colorectal cancer metastatic to the liver using the Sleeping Beauty Transposon SystemPreclinical evaluation of cyclin dependent kinase 11 and casein kinase 2 survival kinases as RNA interference targets for triple negative breast cancer therapyNanoencapsulated anti-CK2 small molecule drug or siRNA specifically targets malignant cancer but not benign cellsHyperactive piggyBac gene transfer in human cells and in vivo.Retargeting transposon insertions by the adeno-associated virus Rep protein.Efficient sleeping beauty DNA transposition from DNA minicircles.CK2 targeted RNAi therapeutic delivered via malignant cell-directed tenfibgen nanocapsule: dose and molecular mechanisms of response in xenograft prostate tumors.β-globin matrix attachment region improves stable genomic expression of the Sleeping Beauty transposon.Engineering of PEDF-Expressing Primary Pigment Epithelial Cells by the SB Transposon System Delivered by pFAR4 Plasmids.The liver works as a school to educate regulatory immune cells.Gene therapy for liver regeneration: experimental studies and prospects for clinical trials.Immunomodulation for inhibitors in hemophilia A: the important role of Treg cellsThe expanding universe of transposon technologies for gene and cell engineering.Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies.Gene editing technology as an approach to the treatment of liver diseases.High Efficiency Ex Vivo Gene Transfer to Primary Murine B Cells Using Plasmid or Viral VectorsHyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy.Advances in Overcoming Immune Responses following Hemophilia Gene Therapy.Engineered Hematopoietic Stem Cells as Therapeutics for Hemophilia A.Preclinical and clinical advances in transposon-based gene therapy.
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P2860
Nanocapsule-delivered Sleeping Beauty mediates therapeutic Factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice.
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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 08 June 2009
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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
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
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Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
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type
label
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
@en
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
@nl
prefLabel
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
@en
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
@nl
P2093
P2860
P921
P356
P1476
Nanocapsule-delivered Sleeping ...... al cells of hemophilia A mice.
@en
P2093
Alycia A Trossen
Betsy T Kren
Brenda M Diethelm-Okita
Clifford J Steer
Gretchen M Unger
Lucas Sjeklocha
Mark T Reding
Vicci Korman
P2860
P304
P356
10.1172/JCI34332
P407
P577
2009-06-08T00:00:00Z