Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.
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Impaired neutralising antibody formation and high transduction efficacy after isolated hepatic perfusion with adenoviral vectors.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype.Gene therapy for haemophilia.Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells.Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trialInduction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.Immune responses to adeno-associated virus and its recombinant vectors.Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challengesSafe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primatesAAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.Prospects for gene therapy of haemophilia.Systemic protein delivery by muscle-gene transfer is limited by a local immune response.Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liverGene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development.Preclinical and clinical gene therapy for haemophilia.Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses.Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.Tolerance induction by viral in vivo gene transferPrediction of adeno-associated virus neutralizing antibody activity for clinical application.Gene therapy for treatment of inherited haematological disorders.Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporineAAV-mediated gene transfer of human pigment epithelium-derived factor inhibits Lewis lung carcinoma growth in mice.Novel vectors for in vivo gene delivery to vascular tissue.Enhancing transduction of the liver by adeno-associated viral vectors.Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivoAAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical modelsRecombinant adeno-associated virus serotype 9 in a mouse model of atherosclerosis: Determination of the optimal expression time in vivo.Our journey to successful gene therapy for hemophilia B.Stable and high-level production of recombinant Factor IX in human hepatic cell line.Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.Nerve growth factor gene therapy using adeno-associated viral vectors prevents cardiomyopathy in type 1 diabetic mice.Enhancement of reporter gene detection sensitivity by insertion of specific mini-peptide-coding sequencesEngineering adeno-associated virus 2 vectors for targeted gene delivery to atherosclerotic lesions.Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.Vps33b is crucial for structural and functional hepatocyte polarity.Performance of AAV8 vectors expressing human factor IX from a hepatic-selective promoter following intravenous injection into rats.
P2860
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P2860
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.
description
2001 nî lūn-bûn
@nan
2001年の論文
@ja
2001年学术文章
@wuu
2001年学术文章
@zh
2001年学术文章
@zh-cn
2001年学术文章
@zh-hans
2001年学术文章
@zh-my
2001年学术文章
@zh-sg
2001年學術文章
@yue
2001年學術文章
@zh-hant
name
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@en
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@nl
type
label
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@en
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@nl
prefLabel
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@en
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@nl
P2093
P356
P1433
P1476
Factors influencing in vivo tr ...... sing the human factor IX cDNA.
@en
P2093
P304
P356
10.1182/BLOOD.V97.5.1258
P407
P577
2001-03-01T00:00:00Z