檢索結果 - Louise R. Rodino‐Klapac

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  1. 1
    Duchenne muscular dystrophy: CRISPR/Cas9 treatment

    Duchenne muscular dystrophy: CRISPR/Cas9 treatment 由 Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2016
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  2. 2
    Update on the Treatment of Duchenne Muscular Dystrophy

    Update on the Treatment of Duchenne Muscular Dystrophy 由 Louise R. Rodino‐Klapac, Jerry R. Mendell, Zarife Sahenk

    出版 2013
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  3. 3
    Emerging drugs for Duchenne muscular dystrophy

    Emerging drugs for Duchenne muscular dystrophy 由 Vinod Malik, Louise R. Rodino‐Klapac, Jerry R. Mendell

    出版 2012
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  4. 4
    Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

    Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy 由 Vinod Malik, Louise R. Rodino‐Klapac, Laurence Viollet, Jerry R. Mendell

    出版 2010
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  5. 5
    Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

    Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy 由 Samiah Al-Zaidy, Zarife Sahenk, Louise R. Rodino‐Klapac, Brian K. Kaspar, Jerry R. Mendell

    出版 2015
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  6. 6
    Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

    Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice 由 Eric R. Pozsgai, Danielle A. Griffin, Kristin N. Heller, Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2017
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  7. 7
    Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

    Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease 由 Louise R. Rodino‐Klapac, Amanda M. Haidet, Janaiah Kota, Chalonda R. Handy, Brian K. Kaspar, Jerry R. Mendell

    出版 2009
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  8. 8
    Gene therapy for muscular dystrophy: Lessons learned and path forward

    Gene therapy for muscular dystrophy: Lessons learned and path forward 由 Jerry R. Mendell, Louise R. Rodino‐Klapac, Zarife Sahenk, Vinod Malik, Brian K. Kaspar, Christopher M. Walker, K. Reed Clark

    出版 2012
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  9. 9
    Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

    Clinical development on the frontier: gene therapy for duchenne muscular dystrophy 由 Damon R. Asher, Khampaseuth Thapa, Sachi D. Dharia, Navid Khan, Rachael A. Potter, Louise R. Rodino‐Klapac, Jerry R. Mendell

    出版 2020
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  10. 10
    Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy

    Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the <i>mdx</i> Mouse Model of Duchenne Muscular Dystrophy 由 Rachael A. Potter, Danielle A. Griffin, Kristin N. Heller, Ellyn L. Peterson, Emma K. Clark, Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2021
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  11. 11
    Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74

    Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74 由 Rachael A. Potter, Ellyn L. Peterson, Danielle Griffin, Grace Cooper Olson, Sarah Lewis, Kyle Cochran, Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2024
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  12. 12
    A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

    A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy 由 Louise R. Rodino‐Klapac, Paul M.L. Janssen, Chrystal L. Montgomery, Brian D. Coley, Louis G. Chicoine, K. Reed Clark, Jerry R. Mendell

    出版 2007
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  13. 13
    Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

    Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations 由 Jerry R. Mendell, Anne M. Connolly, Kelly J. Lehman, Danielle A. Griffin, Sohrab Khan, Sachi D. Dharia, Lucía Quintana‐Gallardo, Louise R. Rodino‐Klapac

    出版 2022
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  14. 14
    AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

    AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy 由 Zarife Sahenk, Gloria Galloway, K. Reed Clark, Vinod Malik, Louise R. Rodino‐Klapac, Brian K. Kaspar, Lei Chen, Cilwyn Braganza, Chrystal L. Montgomery, Jerry R. Mendell

    出版 2013
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  15. 15
    mdx Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits than Do mdx Mice

    mdx Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits than Do mdx Mice 由 Nicholas Beastrom, Haiyan Lu, Allison Macke, Benjamin D. Canan, Eric K. Johnson, Christopher Penton, Brian K. Kaspar, Louise R. Rodino‐Klapac, Lan Zhou, Paul M.L. Janssen, Federica Montanaro

    出版 2011
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  16. 16
    Defective membrane fusion and repair in<i>Anoctamin5</i>-deficient muscular dystrophy

    Defective membrane fusion and repair in<i>Anoctamin5</i>-deficient muscular dystrophy 由 Danielle A. Griffin, R.W. Johnson, Jarred M. Whitlock, Eric R. Pozsgai, Kristin N. Heller, William E. Grose, W. David Arnold, Zarife Sahenk, H. Criss Hartzell, Louise R. Rodino‐Klapac

    出版 2016
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  17. 17
    Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

    Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD 由 Lindsay M. Wallace, Nizar Y. Saad, Nettie K. Pyne, Allison M. Fowler, Jocelyn O. Eidahl, Jacqueline S. Domire, Danielle A. Griffin, Adam Herman, Zarife Sahenk, Louise R. Rodino‐Klapac, Scott Q. Harper

    出版 2017
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  18. 18
    Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

    Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer 由 William E. Grose, K. Reed Clark, Danielle Griffin, Vinod Malik, Kimberly M. Shontz, Chrystal L. Montgomery, Sarah Lewis, Robert H. Brown, Paul M.L. Janssen, Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2012
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  19. 19
    Overexpression of <i>Galgt2</i> in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

    Overexpression of <i>Galgt2</i> in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice 由 Paul T. Martin, Rui Xu, Louise R. Rodino‐Klapac, Elaine Oglesbay, Marybeth Camboni, Chrystal L. Montgomery, Kim Shontz, Louis G. Chicoine, K. Reed Clark, Zarife Sahenk, Jerry R. Mendell, Paul M.L. Janssen

    出版 2008
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  20. 20
    AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

    AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models 由 Patricia Sondergaard, Danielle A. Griffin, Eric R. Pozsgai, R.W. Johnson, William E. Grose, Kristin N. Heller, Kim Shontz, Chrystal L. Montgomery, Joseph Liu, K. Reed Clark, Zarife Sahenk, Jerry R. Mendell, Louise R. Rodino‐Klapac

    出版 2015
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