Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques.
about
AAV's anatomy: roadmap for optimizing vectors for translational successHost and vector-dependent effects on the risk of germline transmission of AAV vectors.AAV8-mediated gene therapy prevents induced biochemical attacks of acute intermittent porphyria and improves neuromotor function.Hepatic gene transfer as a means of tolerance induction to transgene productsAAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T cellsGene delivery by surface immobilization of plasmid to tissue-engineering scaffolds.Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.Platelets as delivery systems for disease treatmentsGood manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trialLong-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.Characterization of naturally-occurring humoral immunity to AAV in sheep.Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal modelGene therapy progress and prospects: gene therapy for the hemophilias.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 macaquesSafe 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 primatesChitosan-DNA nanoparticles delivered by intrabiliary infusion enhance liver-targeted gene deliveryAAV-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 liverLiver gene therapy: advances and hurdles.Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivoPreclinical and clinical gene therapy for haemophilia.Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.PEG-b-PPA/DNA micelles improve transgene expression in rat liver through intrabiliary infusion.Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques.Optimal haemophilia care versus the reality.Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.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 cyclosporineElectroporation-mediated gene therapy.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 vivoImmunity to adeno-associated virus vectors in animals and humans: a continued challenge.Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors.Adeno-associated virus vector-mediated production of hepatocyte growth factor attenuates liver fibrosis in miceRecombinant AAV as a platform for translating the therapeutic potential of RNA interference.Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina.
P2860
Q33659389-AB016AD3-FFF6-4178-8B89-CB60F02629FAQ33713716-7B008768-E515-48DB-B0FA-D70B7896BE99Q33730418-953064A7-24CC-4FB0-9773-E39F52CA9F04Q33775537-82EC6110-108B-4DE0-8516-BF6E0978C8B0Q33931609-7F5AFD8A-6703-4EE2-AB4F-2C7FA8CC0CFDQ34082344-73AE6A01-DE3D-45BC-94ED-A2F237EE46D9Q34122653-2C426BBE-7283-4C2F-A3ED-3E49CC65BC54Q34348837-3B308A2C-C727-4CE5-B87A-0C05824741D4Q34843808-1114CF9D-EB82-414B-8662-ED3F8D984CD4Q34994506-431BF156-95BE-4364-B87F-889B1089ECF2Q35005408-B5E0DB99-314B-4E89-8070-3E8811175756Q35101904-3E321086-1CCD-4BAA-B566-9C89FCBFA601Q35140784-12C07EDF-6CE4-4658-B7EA-692E9CF8AC57Q35431994-2A9B2CCA-AD86-46E5-9CCA-1BF9C4A68D45Q35568499-04A6ACC3-5681-4A2B-B235-ABE041E27E4FQ35628868-BF348491-0F7A-4AB9-B8C5-63C248DD98FCQ35688220-1D29D897-E102-463E-B0AD-2F9AF912192FQ35733790-5E611303-0235-4CD3-A251-BE7830528A57Q35824841-E001CC57-3003-487D-A3C1-CAE5D2F87BC7Q35847626-84DB8CCA-FE90-4879-A0C0-2B9C3159C5C2Q35848563-DCF594C2-2C5C-49FF-9BD7-B645D13F33ABQ35904443-C5F48590-735E-4BA2-966C-26B725AE3261Q35914452-082CEC2D-1FB6-4FFC-990D-AD3283D3C130Q35915704-7D4B0309-5DB1-4912-83A5-F56DFCFAA555Q36007714-BFA0C097-2978-4B4A-BE10-35A460DF2C31Q36084329-65FAF181-7AF8-4C21-91FC-3101CDC8001DQ36230845-D51DE452-8B68-4A2A-A1E1-FDDD983E1FBBQ36298627-38D81859-C2C9-42A4-BF4B-78590015138CQ36399847-464B098A-01EA-43AB-B459-7C350A1622E9Q36427105-8636AEE6-30F1-4744-9630-50CDCA38A856Q36446948-7B1F3C7D-A8B0-44AD-9432-7B319E635B07Q36479748-7EBF4674-6570-4B41-9BC7-3C924832749DQ36945857-296B983A-CA17-4BB8-B189-2D5BD8A060D2Q37042394-FD509012-1998-4BE5-A5D5-F97254E052AAQ37066326-CC99B27E-5607-452F-AFB9-A4C24FAE3A51Q37127246-98BCDFCB-050B-40F0-AB4D-F23EEB849A14Q37209197-483FA549-46F0-4722-9D86-CD4204F16A02Q37278165-B66FD3F0-C5E8-4E6E-A924-72B00D410FB5Q37696045-6513E823-4723-479D-BC8C-BDCD84D357C0Q37768868-CF6B0CC5-C392-4B93-A4BF-C6D42E1B7BD5
P2860
Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques.
description
2002 nî lūn-bûn
@nan
2002年の論文
@ja
2002年学术文章
@wuu
2002年学术文章
@zh
2002年学术文章
@zh-cn
2002年学术文章
@zh-hans
2002年学术文章
@zh-my
2002年学术文章
@zh-sg
2002年學術文章
@yue
2002年學術文章
@zh-hant
name
Sustained high-level expressio ...... hFIX gene in rhesus macaques.
@en
Sustained high-level expression of human factor IX
@nl
type
label
Sustained high-level expressio ...... hFIX gene in rhesus macaques.
@en
Sustained high-level expression of human factor IX
@nl
prefLabel
Sustained high-level expressio ...... hFIX gene in rhesus macaques.
@en
Sustained high-level expression of human factor IX
@nl
P2093
P1433
P1476
Sustained high-level expressio ...... hFIX gene in rhesus macaques.
@en
P2093
Alexander Nikanorov
Amit C Nathwani
Andrew M Davidoff
Arthur W Nienhuis
Catherine Y C Ng
Clive Slaughter
Elio F Vanin
Fredric A Hoffer
Hideki Hanawa
Jay N Lozier
P304
P356
10.1182/BLOOD-2002-02-0589
P407
P577
2002-09-01T00:00:00Z