Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.

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DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B.Serum exosomes can restore cellular function in vitro and be used for diagnosis in dysferlinopathy.Identification of Novel Antisense-Mediated Exon Skipping Targets in DYSF for Therapeutic Treatment of Dysferlinopathy

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Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.

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Structure-Based Designed Nano- ...... Dysferlinopathy in BLA/J Mice.

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Structure-Based Designed Nano- ...... Dysferlinopathy in BLA/J Mice.

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Structure-Based Designed Nano- ...... Dysferlinopathy in BLA/J Mice.

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Structure-Based Designed Nano- ...... Dysferlinopathy in BLA/J Mice.

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Catherine Dial

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Laura Conatser

http://www.w3.org/2001/XMLSchema#string

Matthew L Hirsch

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Nadia Nagy

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R Bryan Sutton

http://www.w3.org/2001/XMLSchema#string

Telmo Llanga

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P2860

Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer

Lack of correlation between outcomes of membrane repair assay and correction of dystrophic changes in experimental therapeutic strategy in dysferlinopathy

Defective membrane repair in dysferlin-deficient muscular dystrophy

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

A single C2 domain from synaptotagmin I is sufficient for high affinity Ca2+/phospholipid binding

Quantitation of the calcium and membrane binding properties of the C2 domains of dysferlin

Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences.

Generation of a synthetic mammalian promoter library by modification of sequences spacing transcription factor binding sites.

AAV recombineering with single strand oligonucleotides.

Characterization of genome integrity for oversized recombinant AAV vector.

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scholarly article

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10.1016/J.YMTHE.2017.05.013

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2017-06-16T00:00:00Z

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28629822

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5589059

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Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.

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P2860

P2860

Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice.

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P1476

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