Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?
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Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B miceAnimal models of hemophiliaTranslational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogsThermal scalpel to target cancer.Clinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons.Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer.Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.Optimization of adenoviral vector-mediated transgene expression in the canine brain in vivo, and in canine glioma cells in vitro.Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens.Central venous access devices in patients with hemophilia.Optimal haemophilia care versus the reality.Ethical issues in haemophilia.Treatment of Hemophilia A in Utero and Postnatally using Sheep as a Model for Cell and Gene DeliveryEnhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.Immunological aspects of inhibitor development.Haemophilia A: molecular insights.Staunch protections: the ethics of haemophilia gene transfer research.Advances in microRNAs: implications for gene therapists.In vivo models of haemophilia - status on current knowledge of clinical phenotypes and therapeutic interventions.Successful treatment of canine hemophilia by continuous expression of canine FVIIa.Long-term transduction of miniature pig parotid glands using serotype 2 adeno-associated viral vectorsSupplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies.Diverse IgG subclass responses to adeno-associated virus infection and vector administration
P2860
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P2860
Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?
description
2005 nî lūn-bûn
@nan
2005年の論文
@ja
2005年論文
@yue
2005年論文
@zh-hant
2005年論文
@zh-hk
2005年論文
@zh-mo
2005年論文
@zh-tw
2005年论文
@wuu
2005年论文
@zh
2005年论文
@zh-cn
name
Gene transfer for hemophilia: ...... safely translated to patients?
@ast
Gene transfer for hemophilia: ...... safely translated to patients?
@en
type
label
Gene transfer for hemophilia: ...... safely translated to patients?
@ast
Gene transfer for hemophilia: ...... safely translated to patients?
@en
prefLabel
Gene transfer for hemophilia: ...... safely translated to patients?
@ast
Gene transfer for hemophilia: ...... safely translated to patients?
@en
P2860
P1476
Gene transfer for hemophilia: ...... safely translated to patients?
@en
P2093
P2860
P304
P356
10.1111/J.1538-7836.2005.01460.X
P577
2005-08-01T00:00:00Z