Signs of Progress in Gene Therapy for Muscular Dystrophy Also Warrant Caution

Por Stedman, Hansell H, Byrne, Barry J
Publicado em 2012

Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes

Por VanBelzen, D. Jake, Malik, Alock S., Henthorn, Paula S., Kornegay, Joe N., Stedman, Hansell H.
Publicado no Mol Ther Methods Clin Dev (2016)

Suite of clinically relevant functional assays to address therapeutic efficacy and disease mechanism in the dystrophic mdx mouse

Por Song, Yafeng, Rosenblum, Shira T., Morales, Leon, Petrov, Mihail, Greer, Christopher, Globerman, Samantha, Stedman, Hansell H.
Publicado no J Appl Physiol (1985) (2017)

Cardiac Gene Therapy: Optimization of Gene Delivery Techniques In Vivo

Por Katz, Michael G., Swain, JaBaris D., White, Jennifer D., Low, David, Stedman, Hansell, Bridges, Charles R.
Publicado em 2010

Translational Data from Adeno-Associated Virus-Mediated Gene Therapy of Hemophilia B in Dogs

Por Nichols, Timothy C., Whitford, Margaret H., Arruda, Valder R., Stedman, Hansell H., Kay, Mark A., High, Katherine A.
Publicado no Hum Gene Ther Clin Dev (2015)

A Pharmacokinetic Analysis of Molecular Cardiac Surgery With Recirculation Mediated Delivery of βARKct Gene Therapy: Developing a Quantitative Definition of the Therapeutic Window

Por FARGNOLI, ANTHONY S., KATZ, MICHAEL G., YARNALL, CHARLES, SUMAROKA, MARINA V., STEDMAN, HANSELL, RABINOWITZ, JOSEPH J., KOCH, WALTER J., BRIDGES, CHARLES R.
Publicado em 2011

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Por Arruda, Valder R., Stedman, Hansell H., Nichols, Timothy C., Haskins, Mark E., Nicholson, Matthew, Herzog, Roland W., Couto, Linda B., High, Katherine A.
Publicado em 2005

Myosin heavy chain and physiological adaptation of the rat diaphragm in elastase-induced emphysema

Por Kim, Dong Kwan, Zhu, Jianliang, Kozyak, Benjamin W, Burkman, James M, Rubinstein, Neal A, Lankford, Edward B, Stedman, Hansell H, Nguyen, Taitan, Levine, Sanford, Shrager, Joseph B
Publicado em 2003

Cardiac Surgical Delivery of the Sarcoplasmic Reticulum Calcium ATPase Rescues Myocytes in Ischemic Heart Failure

Por Fargnoli, Anthony S., Katz, Michael G., Yarnall, Charles, Isidro, Alice, Petrov, Michael, Steuerwald, Nury, Ghosh, Sriparna, Richardville, Kyle C., Hillesheim, Richard, Williams, Richard D., Kohlbrenner, Erik, Stedman, Hansell H., Hajjar, Roger J., Bridges, Charles R.
Publicado em 2013

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

Por Haurigot, Virginia, Mingozzi, Federico, Buchlis, George, Hui, Daniel J, Chen, Yifeng, Basner-Tschakarjan, Etiena, Arruda, Valder R, Radu, Antoneta, Franck, Helen G, Wright, J Fraser, Zhou, Shangzhen, Stedman, Hansell H, Bellinger, Dwight A, Nichols, Timothy C, High, Katherine A
Publicado em 2010

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Por Arruda, Valder R., Stedman, Hansell H., Haurigot, Virginia, Buchlis, George, Baila, Stefano, Favaro, Patricia, Chen, Yifeng, Franck, Helen G., Zhou, Shangzhen, Wright, J. Fraser, Couto, Linda B., Jiang, Haiyan, Pierce, Glenn F., Bellinger, Dwight A., Mingozzi, Federico, Nichols, Timothy C., High, Katherine A.
Publicado em 2010

Perspectives on Best Practices for Gene Therapy Programs

Por Cheever, Thomas R., Berkley, Dale, Braun, Serge, Brown, Robert H., Byrne, Barry J., Chamberlain, Jeffrey S., Cwik, Valerie, Duan, Dongsheng, Federoff, Howard J., High, Katherine A., Kaspar, Brian K., Klinger, Katherine W., Larkindale, Jane, Lincecum, John, Mavilio, Fulvio, McDonald, Cheryl L., McLaughlin, James, Weiss McLeod, Bonnie, Mendell, Jerry R., Nuckolls, Glen, Stedman, Hansell H., Tagle, Danilo A., Vandenberghe, Luk H., Wang, Hao, Wernett, Pamela J., Wilson, James M., Porter, John D., Gubitz, Amelie K.
Publicado no Hum Gene Ther (2015)