Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.

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Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients.

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Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.

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2017 nî lūn-bûn

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2017年の論文

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2017年論文

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2017年論文

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2017年論文

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Systemic AAV-Mediated β-Sarcog ...... ional Deficits in LGMD2E Mice.

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Systemic AAV-Mediated β-Sarcog ...... ional Deficits in LGMD2E Mice.

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Systemic AAV-Mediated β-Sarcog ...... ional Deficits in LGMD2E Mice.

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P1476

Systemic AAV-Mediated β-Sarcog ...... ional Deficits in LGMD2E Mice.

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Danielle A Griffin

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Eric R Pozsgai

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Jerry R Mendell

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Kristin N Heller

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Louise R Rodino-Klapac

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P2860

Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

Disruption of heart sarcoglycan complex and severe cardiomyopathy caused by beta sarcoglycan mutations

Genomic screening for beta-sarcoglycan gene mutations: missense mutations may cause severe limb-girdle muscular dystrophy type 2E (LGMD 2E)

Disruption of the beta-sarcoglycan gene reveals pathogenetic complexity of limb-girdle muscular dystrophy type 2E

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

Beta-sarcoglycan (A3b) mutations cause autosomal recessive muscular dystrophy with loss of the sarcoglycan complex

The clinical spectrum of sarcoglycanopathies

Prevention of cardiomyopathy in mouse models lacking the smooth muscle sarcoglycan-sarcospan complex

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.

Diastolic dysfunction and cardiac troponin I decrease in aging hearts.

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scholarly article

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10.1016/J.YMTHE.2017.02.013

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2017-03-09T00:00:00Z

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Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.

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P2860

P2860

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.

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P1433

P1476

P2093

P2860

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P356

P433

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P698

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P2093

P2860

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P356

P433

P478

P577

P698

P932