Safe 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 primates
about
Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genomeClinical development of gene therapy: results and lessons from recent successesAdeno-associated Virus as a Mammalian DNA VectorDevelopments in the treatment of hemophilia B: focus on emerging gene therapyThe gene therapy journey for hemophilia: are we there yet?Structural Insights into Adeno-Associated Virus Serotype 5Gene therapy in an era of emerging treatment options for hemophilia BLong-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transferTreatment of diabetes and long-term survival after insulin and glucokinase gene therapy.Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders.AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.Progress towards gene therapy for haemophilia B.Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.AAV5-mediated gene transfer to the parotid glands of non-human primatesEradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft modelB-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case StudyLong-term safety and efficacy of factor IX gene therapy in hemophilia B.Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trialPhenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus 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.A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates.Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency.The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver.Employing 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.Recombinant AAV-directed gene therapy for type I glycogen storage diseases.Adeno-associated viral vector serotype 5 poorly transduces liver in rat modelsSafety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal modelEvaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challengesStable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaquesSystemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expressionHepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector.AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primatesImpact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors
P2860
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P2860
Safe 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 primates
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
2006年论文
@zh
2006年论文
@zh-cn
name
Safe and efficient transductio ...... human FIX in nonhuman primates
@ast
Safe and efficient transductio ...... human FIX in nonhuman primates
@en
type
label
Safe and efficient transductio ...... human FIX in nonhuman primates
@ast
Safe and efficient transductio ...... human FIX in nonhuman primates
@en
prefLabel
Safe and efficient transductio ...... human FIX in nonhuman primates
@ast
Safe and efficient transductio ...... human FIX in nonhuman primates
@en
P2093
P2860
P1433
P1476
Safe and efficient transductio ...... human FIX in nonhuman primates
@en
P2093
Amit C Nathwani
Andrew M Davidoff
Catherine Y C Ng
Edward G D Tuddenham
Elaine Gray
Jenny McIntosh
John T Gray
Junfang Zhou
Melanie Cochrane
Yunyu Spence
P2860
P304
P356
10.1182/BLOOD-2006-03-010181
P407
P577
2006-11-07T00:00:00Z