A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence
about
Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Hip region muscular dystrophy and emergence of motor deficits in dysferlin-deficient Bla/J mice.Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts.DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Triple Vectors Expand AAV Transfer Capacity in the Retina.Treatment with Recombinant Human MG53 Protein Increases Membrane Integrity in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.
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P2860
A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence
description
2015 nî lūn-bûn
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2015年の論文
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2015年学术文章
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2015年学术文章
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2015年学术文章
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2015年学术文章
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2015年学术文章
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name
A comparison of AAV strategies ...... 2 kb Dysferlin coding sequence
@en
type
label
A comparison of AAV strategies ...... 2 kb Dysferlin coding sequence
@en
prefLabel
A comparison of AAV strategies ...... 2 kb Dysferlin coding sequence
@en
P2093
P2860
P356
P1476
A comparison of AAV strategies ...... 2 kb Dysferlin coding sequence
@en
P2093
Carinne Roudaut
Isabelle Richard
Karine Charton
Marina Pryadkina
Matthew L Hirsch
Nathalie Bourg
P2860
P356
10.1038/MTM.2015.9
P577
2015-03-25T00:00:00Z