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Skeletal Muscle|August 7, 2013
Increased sphingosine-1-phosphate improves muscle regeneration in acutely injured mdx miceNicholas Ieronimakis, Mario Pantoja, Aislinn L Hays, et al.Nature Medicine|July 5, 2006
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic micePaul Gregorevic, James M Allen, Elina Minami, et al.JACC. Basic to Translational Science|May 30, 2017
Translation of Cardiac Myosin Activation with 2-deoxy-ATP to Treat Heart Failure via an Experimental Ribonucleotide Reductase-Based Gene TherapyKassandra S Thomson, Guy L Odom, Charles E Murry, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|June 14, 2012
Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapiesZejing Wang, Rainer Storb, Christine L Halbert, et al.Molecular Therapy. Methods & Clinical Development|June 26, 2023
Efficacy and muscle safety assessment of fukutin-related protein gene therapyHalli Benasutti, Joseph W Maricelli, Jane Seto, et al.Progress in Molecular Biology and Translational Science|December 6, 2011
Animal models of muscular dystrophyRainer Ng, Glen B Banks, John K Hall, et al.Nature Communications|March 7, 2013
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cellsAntonio Filareto, Sarah Parker, Radbod Darabi, et al.Human Gene Therapy|June 7, 2007
Herpes simplex virus VP22 enhances adenovirus-mediated microdystrophin gene transfer to skeletal muscles in dystrophin-deficient (mdx) miceFu Xiong, Shaobo Xiao, Funing Peng, et al.Nature Medicine|March 5, 2002
Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophyScott Q Harper, Michael A Hauser, Christiana DelloRusso, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|January 12, 2002
Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vectorP Seshidhar Reddy, Kiran Sakhuja, Shanthi Ganesh, et al.Pageof 16