Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.
about
Gene therapy: prospects for glycolipid storage diseases.Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry DiseaseCharacterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.Depletion of globosides and isoglobosides fully reverts the morphologic phenotype of Fabry diseaseLong-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer.Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives.A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transductionAdvances in the management of Anderson-Fabry disease: enzyme replacement therapy.Agalsidase alfa--a preparation for enzyme replacement therapy in Anderson-Fabry disease.α-Galactosidase A expressed in the salivary glands partially corrects organ biochemical deficits in the fabry mouse through endocrine traffickingEnzyme replacement therapy for Fabry disease: some answers but more questionsLong-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transferCombination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brainImmune responses to adeno-associated virus and its recombinant vectors.Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities.Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann-Pick mouse.Production of recombinant beta-hexosaminidase A, a potential enzyme for replacement therapy for Tay-Sachs and Sandhoff diseases, in the methylotrophic yeast Ogataea minutaNovel therapeutic targets for the treatment of Fabry disease.Protective effect of recombinant adeno-associated virus 2/8-mediated gene therapy from the maternal hyperphenylalaninemia in offsprings of a mouse model of phenylketonuria.Protection of a ceramide synthase 2 null mouse from drug-induced liver injury: role of gap junction dysfunction and connexin 32 mislocalizationViral vectors for vascular gene therapyAAV-directed muscular dystrophy gene therapy.Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors.Neurological features of Fabry disease: clinical, pathophysiological aspects and therapy.Gene therapy for fabry disease: a review of the literature.Fabry disease: experience of screening dialysis patients for Fabry disease.Fabry Disease: Recognition, Diagnosis, and Treatment of Neurological Features.Promise of adeno-associated virus as a gene therapy vector for cardiovascular diseases.Feasibility of generating adeno-associated virus packaging cell lines containing inducible adenovirus helper genes.Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy.Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.Delivery of glucose-6-phosphatase in a canine model for glycogen storage disease, type Ia, with adeno-associated virus (AAV) vectors.Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease.Efficient correction of Fabry mice and patient cells mediated by lentiviral transduction of hematopoietic stem/progenitor cells.Prospects and problems of gene therapy: an update.Globotriaosylceramide leads to K(Ca)3.1 channel dysfunction: a new insight into endothelial dysfunction in Fabry disease.
P2860
Q30499839-65650A71-C0F2-46E9-9A46-4DA2AC8CB5C4Q33752595-F7BF8613-C618-4871-B7D9-0A9EB71103DDQ33818578-8DAA8603-64B3-43FE-94AC-6F889B1EBDCFQ34161415-C0C5091A-024B-456D-B8A5-C06AC4D57F3CQ34294988-D2D517A3-924A-4DFD-A27A-3E81FE7A458EQ34325086-119118D8-B445-4A64-95DC-0BC431F91C31Q34354096-7FAF05ED-53F5-4661-8FBD-76D3D2EDC761Q34356203-761EC328-789E-463E-990F-63CF885B0611Q34560688-115D895E-4732-46E9-A962-32531A3581EEQ34660894-0475C054-C26D-481E-ADBC-A3B9189373FEQ34671627-B2B1E6D3-A2B6-4C1F-9D5E-E21B668FEC6EQ34703815-ADF52448-3719-483A-81DD-7630FF30D336Q34870564-D7AA0EEE-0AE3-475E-90CC-8EFA88FFADCAQ34982831-46CDE96A-116A-4E1B-A124-91F1B92D2FC8Q35000819-2820217F-AB6F-49AB-BFFF-4CCCF166D55BQ35131229-8478CD66-376A-41A6-AFD8-67D8F9B70450Q35186114-FE8B8216-AE38-4C7D-AB60-2936B23F905BQ35238264-AF2A400C-A2B0-459D-8CF0-5399E92A4037Q35750005-DAAEB96F-15BF-4D4D-BB1F-10E925204392Q35803520-36BEBAA2-EB33-43EA-BDCD-F88FC3B3FBD4Q35946763-3E4B3339-5B30-4388-B37B-634C99C7A3EFQ36822452-DA153C37-F8B1-4CBE-B95D-768CD044BE3DQ36966120-7D3A205A-D065-4520-B780-AF6D318C78F9Q37312126-C448E272-B8DC-4B29-B1E1-D8A477AFE88EQ37569631-789F35B6-01A7-423E-BCED-63C2AB26EA6BQ37687423-67F37355-875A-498B-8D9F-316FE6EE481EQ37695619-98DF21E1-F275-4392-A39B-321489816CC0Q37994740-595E0EBF-8322-4D01-A8B2-2CC8741E3140Q38098070-357E7037-FC2D-4F17-9ED6-186916C501E8Q38159935-6E5E0694-C522-46FA-BA1F-9D5375B206FBQ38844401-40BD41D7-ABBE-4CBA-8F72-7C4469E81456Q39356341-96BAE1AD-CBA2-462E-B87B-FF556050C562Q39682511-A6CB4366-46B1-4487-88D5-3BE2759FAD82Q41619077-3B20ED2F-BE79-4628-9D3F-36F63054C9A5Q41999615-E32F7B95-7C06-4C94-8A98-054A3A27EB73Q44052705-27543281-7896-4323-9DBD-AE8E6DED7922Q45856748-1C79E818-DA16-4A89-B775-70F88CB234D0Q45861979-28FBE8A4-ABF5-4B20-AE9C-C3AD94281280Q45884593-29E8919E-B697-459C-97FB-9183DD7FD6E0Q53453652-F6A22B5F-D4E6-4DC8-8DEF-C4AE42E39739
P2860
Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.
description
2001 nî lūn-bûn
@nan
2001 թուականի Փետրուարին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի փետրվարին հրատարակված գիտական հոդված
@hy
2001年の論文
@ja
2001年論文
@yue
2001年論文
@zh-hant
2001年論文
@zh-hk
2001年論文
@zh-mo
2001年論文
@zh-tw
2001年论文
@wuu
name
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@ast
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@en
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@nl
type
label
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@ast
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@en
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@nl
prefLabel
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@ast
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@en
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@nl
P2093
P2860
P356
P1476
Adeno-associated viral vector- ...... multiple organs of Fabry mice.
@en
P2093
Gelderman MP
Tirumalai K
P2860
P304
P356
10.1073/PNAS.051634498
P407
P577
2001-02-01T00:00:00Z