Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.
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Targeted approaches to induce immune tolerance for Pompe disease therapyEmerging and future therapies for hemophiliaGene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translationStructural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9Terminal differentiation of cardiac and skeletal myocytes induces permissivity to AAV transduction by relieving inhibition imposed by DNA damage response proteinsCollaboration for rare disease drug discovery researchBAAV transcytosis requires an interaction with beta-1-4 linked- glucosamine and gp96Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transferAAV's anatomy: roadmap for optimizing vectors for translational successIntravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function.HIV-1 lentiviral vector immunogenicity is mediated by Toll-like receptor 3 (TLR3) and TLR7.A NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (AAV)-BASED RANDOM PEPTIDE DISPLAY LIBRARY SYSTEM: INFECTION-DEFECTIVE AAV1.9-3 AS A NOVEL DETARGETED PLATFORM FOR VECTOR EVOLUTION.Combined paracrine and endocrine AAV9 mediated expression of hepatocyte growth factor for the treatment of renal fibrosisAdeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.Repression of cardiac hypertrophy by KLF15: underlying mechanisms and therapeutic implications.Hyperactive sleeping beauty transposase enables persistent phenotypic correction in mice and a canine model for hemophilia B.Adeno-associated virus: a key to the human genome?Dendritic cell-directed vaccination with a lentivector encoding PSCA for prostate cancer in miceEfficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.In vitro evaluation of a double-stranded self-complementary adeno-associated virus type2 vector in bone marrow stromal cells for bone healing.The AAV9 receptor and its modification to improve in vivo lung gene transfer in miceEmploying a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction.Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities.Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines.Intracellular transport of recombinant adeno-associated virus vectors.Genome-wide RNAi screening identifies host restriction factors critical for in vivo AAV transductionMuscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.Identification of the galactose binding domain of the adeno-associated virus serotype 9 capsidCopackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Gene therapy, bioengineered clotting factors and novel technologies for hemophilia treatment.Novel vectors for in vivo gene delivery to vascular tissue.Optimized adeno-associated virus 8 produces hepatocyte-specific Cre-mediated recombination without toxicity or affecting liver regeneration.Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice.Meeting report: ninth and tenth workshops of the European Paediatric Network for Haemophilia Management (PedNet).Recombinant adeno-associated virus transduction and integration.The state of the art of adeno-associated virus-based vectors in gene therapy.Immune responses to AAV vectors: overcoming barriers to successful gene therapy
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P2860
Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年学术文章
@wuu
2006年学术文章
@zh
2006年学术文章
@zh-cn
2006年学术文章
@zh-hans
2006年学术文章
@zh-my
2006年学术文章
@zh-sg
2006年學術文章
@yue
2006年學術文章
@zh-hant
name
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@en
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@nl
type
label
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@en
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@nl
prefLabel
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@en
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@nl
P2093
P2860
P921
P1476
Efficacy and safety of adeno-a ...... for hemophilia B gene therapy.
@en
P2093
Acosta-Sanchez A
DE Waele L
Gillijns V
Vandendriessche T
P2860
P356
10.1111/J.1538-7836.2006.02220.X
P577
2006-09-26T00:00:00Z