Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.
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Therapies for the bone in mucopolysaccharidosesSimilar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage diseaseGene therapy for lysosomal storage disorders: a good start.Therapies of mucopolysaccharidosis IVA (Morquio A syndrome).Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial.A review of gene therapy in canine and feline models of lysosomal storage disorders.Diagnostic and treatment strategies in mucopolysaccharidosis VI.Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.Gene delivery strategies for the treatment of mucopolysaccharidoses.Emerging drugs for the treatment of mucopolysaccharidoses.α- L-iduronidase gene-based therapy using the phiC31 system to treat mucopolysaccharidose type I mice.Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI.Gene therapy for Mucopolysaccharidoses.Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice
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P2860
Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@ast
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@en
type
label
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@ast
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@en
prefLabel
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@ast
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@en
P2093
P2860
P356
P1433
P1476
Gene therapy for mucopolysacch ...... pre-existing immunity to AAV8.
@en
P2093
Alberto Auricchio
Gabriella Cotugno
James M Wilson
Mark Haskins
Pamela Claudiani
Patricia O'Donnell
Rita Ferla
Roberto Calcedo
Thomas O'Malley
P2860
P304
P356
10.1089/HUM.2012.179
P577
2013-01-30T00:00:00Z