Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs von Mack, David L., Poulard, Karine, Goddard, Melissa A., Latournerie, Virginie, Snyder, Jessica M., Grange, Robert W., Elverman, Matthew R., Denard, Jérôme, Veron, Philippe, Buscara, Laurine, Le Bec, Christine, Hogrel, Jean-Yves, Brezovec, Annie G., Meng, Hui, Yang, Lin, Liu, Fujun, O’Callaghan, Michael, Gopal, Nikhil, Kelly, Valerie E., Smith, Barbara K., Strande, Jennifer L., Mavilio, Fulvio, Beggs, Alan H., Mingozzi, Federico, Lawlor, Michael W., Buj-Bello, Ana, Childers, Martin K.

Volltext

Long‐term effects of systemic gene therapy in a canine model of myotubular myopathy von Elverman, Matthew, Goddard, Melissa A., Mack, David, Snyder, Jessica M., Lawlor, Michael W., Meng, Hui, Beggs, Alan H., Buj‐Bello, Ana, Poulard, Karine, Marsh, Anthony P., Grange, Robert W., Kelly, Valerie E., Childers, Martin K.

Volltext

Gene therapy in monogenic congenital myopathies von Guan, Xuan, Goddard, Melissa A., Mack, David L., Childers, Martin K.

Volltext

Gait characteristics in a canine model of X-linked myotubular myopathy von Goddard, Melissa A, Burlingame, Emily, Beggs, Alan H, Buj-Bello, Anna, Childers, Martin K, Marsh, Anthony P, Kelly, Valerie E

Volltext

Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis von Taglietti, Valentina, Kefi, Kaouthar, Bronisz-Budzyńska, Iwona, Mirciloglu, Busra, Rodrigues, Mathilde, Cardone, Nastasia, Coulpier, Fanny, Periou, Baptiste, Gentil, Christel, Goddard, Melissa, Authier, François-Jérôme, Pietri-Rouxel, France, Malfatti, Edoardo, Lafuste, Peggy, Tiret, Laurent, Relaix, Frederic

Volltext

Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myo... von Goddard, Melissa A, Mack, David L, Czerniecki, Stefan M, Kelly, Valerie E, Snyder, Jessica M, Grange, Robert W, Lawlor, Michael W, Smith, Barbara K, Beggs, Alan H, Childers, Martin K

Volltext

Respiratory assessment in centronuclear myopathies von Smith, Barbara K., Goddard, Melissa, Childers, Martin K.

Volltext

Long‐term effects of systemic gene therapy in a canine model of myotubular myopathy von Elverman, Matthew, Goddard, Melissa, Mack, David, Snyder, Jessica, Lawlor, Michael, Meng, Hui, Beggs, Alan, Buj-Bello, Ana, Poulard, Karine, Marsh, Anthony, Grange, Robert, Kelly, Valerie, Childers, Martin

Volltext

Duchenne muscular dystrophy trajectory in R-DMDdel52 preclinical rat model identifies COMP as biomarker of fibrosis von Taglietti, Valentina, Kefi, Kaouthar, Bronisz-Budzyńska, Iwona, Mirciloglu, Busra, Rodrigues, Mathilde, Cardone, Nastasia, Coulpier, Fanny, Periou, Baptiste, Gentil, Christel, Goddard, Melissa, Authier, François-Jérôme, Pietri-Rouxel, France, Malfatti, Edoardo, Lafuste, Peggy, Tiret, Laurent, Relaix, Frederic

Volltext

Gait characteristics in a canine model of X-linked myotubular myopathy von Goddard, Melissa, Burlingame, Emily, Beggs, Alan, Buj-Bello, Anna, Childers, Martin, Marsh, Anthony, Kelly, Valerie

Volltext

398. Sustained Expression of a Canine Micro-Dystrophin Lead To Improvement of Limb Muscle Function in Dystrophic Dogs Following Large Scale AAV-Mediated Treatment von Halbert, Christine, Goddard, Melissa, Lee, Donghoon, Mack, David, Butts, Tiffany, Grange, Robert, Johnson, Mackenzie, Storb, Rainer, Miller, Dusty, Chamberlain, Jeffrey, Tapscott, Stephen, Childers, Martin, Wang, Zejing

Volltext

498. Prolonged Benefit from Systemic rAAV8 in a Canine Model of Myotubular Myopathy von Elverman, Matthew, Mack, David, Goddard, Melissa, Snyder, Jessica, Grange, Robert, Poulard, Karine, Latournerie, Virginie, Veron, Philippe, Buscara, Laurine, Bec, Christine Le, Martin, Samia, Mavilio, Fulvio, Mingozzi, Federico, Meng, Hui, Lawlor, Michael, Beggs, Alan, Buj-Bello, Anna, Childers, Martin

Volltext

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs von Mack, David, Poulard, Karine, Goddard, Melissa, Latournerie, Virginie, Snyder, Jessica, Grange, Robert, Elverman, Matthew, Denard, Jérôme, Veron, Philippe, Buscara, Laurine, Le Bec, Christine, Hogrel, Jean-Yves, Brezovec, Annie, Meng, Hui, Yang, Lin, Liu, Fujun, O’callaghan, Michael, Gopal, Nikhil, Kelly, Valerie, Smith, Barbara, Strande, Jennifer, Mavilio, Fulvio, Beggs, Alan, Mingozzi, Federico, Lawlor, Michael, Buj-Bello, Ana, Childers, Martin

Volltext

Muscle function in A canine model of X-linked myotubular myopathy von Grange, Robert W., Doering, Jon, Mitchell, Erin, Holder, Melanie N., Guan, Xuan, Goddard, Melissa, Tegeler, Christopher, Beggs, Alan H., Childers, Martin K.

Volltext

503. Minimally Effective Dose of Systemic AAV8-MTM1 Needed To Prolong Survival and Correct Severe Muscle Pathology in a Canine Model of X-Linked Myotubular Myopathy von Mack, David L., Poulard, Karine, Goddard, Melissa, Snyder, Jessica M., Grange, Robert W., Doering, Jon, Czerniecki, Stefan, Strande, Jennifer L., Latournerie, Virginie, Veron, Philippe, Meng, Hui, Yang, Lin, Liu, Fujun, Le Bec, Christine, Buscara, Laurine, Martin, Samia, Moullier, Philippe, O'Callaghan, Michael, Mingozzi, Federico, Beggs, Alan H., Lawlor, Michael W., Buj-Bello, Anna, Mavilio, Fulvio, Childers, Martin K.

Volltext

Validity of a neurological scoring system for canine X-linked myotubular myopathy von Snyder, Jessica M, Meisner, Allison, Mack, David, Goddard, Melissa, Coulter, Ian T, Grange, Robert, Childers, Martin

Volltext

Validity of a Neurological Scoring System for Canine X-Linked Myotubular Myopathy von Snyder, Jessica M, Meisner, Allison, Mack, David, Goddard, Melissa, Coulter, Ian T, Grange, Robert, Childers, Martin K

Volltext

Erratum to muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myo...

Volltext

Caring for individuals with Type 1 Diabetes Mellitus who restrict and omit insulin for weight control: Evidence-based guidance for healthcare professionals von Goddard, Georgia, Oxlad, Melissa

Volltext

The Genomic Medicine Integrative Research Framework: A Conceptual Framework for Conducting Genomic Medicine Research von Horowitz, Carol R., Orlando, Lori A., Slavotinek, Anne M., Peterson, Josh, Angelo, Frank, Biesecker, Barbara, Bonham, Vence L., Cameron, Linda D., Fullerton, Stephanie M., Gelb, Bruce D., Goddard, Katrina A.B., Hailu, Benyam, Hart, Ragan, Hindorff, Lucia A., Jarvik, Gail P., Kaufman, Dave, Kenny, Eimear E., Knight, Sara J., Koenig, Barbara A., Korf, Bruce R., Madden, Ebony, McGuire, Amy L., Ou, Jeffrey, Wasserstein, Melissa P., Robinson, Mimsie, Leventhal, Howard, Sanderson, Saskia C.

Volltext