Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy

Cora C. Hart; Young il Lee; Jun Xie; Guangping Gao; David W. Hammers; H. Lee Sweeney

doi:10.1101/2022.10.02.510519

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Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy

Cora C. Hart, Young il Lee, Jun Xie, Guangping Gao, David W. Hammers, H. Lee Sweeney

doi: https://doi.org/10.1101/2022.10.02.510519

Cora C. Hart

1Department of Pharmacology & Therapeutics, University of Florida College of Medicine; Gainesville, FL 32610

2Myology Institute, University of Florida College of Medicine; Gainesville, FL 32610

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Young il Lee

1Department of Pharmacology & Therapeutics, University of Florida College of Medicine; Gainesville, FL 32610

2Myology Institute, University of Florida College of Medicine; Gainesville, FL 32610

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Jun Xie

3Horae Gene Therapy Center, University of Massachusetts Medical School, Worchester, MA 01655

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Guangping Gao

3Horae Gene Therapy Center, University of Massachusetts Medical School, Worchester, MA 01655

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David W. Hammers

1Department of Pharmacology & Therapeutics, University of Florida College of Medicine; Gainesville, FL 32610

2Myology Institute, University of Florida College of Medicine; Gainesville, FL 32610

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H. Lee Sweeney

1Department of Pharmacology & Therapeutics, University of Florida College of Medicine; Gainesville, FL 32610

2Myology Institute, University of Florida College of Medicine; Gainesville, FL 32610

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For correspondence: lsweeney@ufl.edu

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Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy

Cora C. Hart, Young il Lee, Jun Xie, Guangping Gao, David W. Hammers, H. Lee Sweeney

bioRxiv 2022.10.02.510519; doi: https://doi.org/10.1101/2022.10.02.510519

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Potential limitations of micro-dystrophin gene therapy for Duchenne muscular dystrophy

Cora C. Hart, Young il Lee, Jun Xie, Guangping Gao, David W. Hammers, H. Lee Sweeney

bioRxiv 2022.10.02.510519; doi: https://doi.org/10.1101/2022.10.02.510519

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