Synthetic Zinc Finger Nuclease Design and Rapid Assembly

Por Osborn, Mark J., DeFeo, Anthony P., Blazar, Bruce R., Tolar, Jakub
Publicado em 2011

Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α--Iduronidase in Mice With Mucopolysaccharidosis Type I

Por Osborn, Mark J, McElmurry, Ron T, Lees, Christopher J, DeFeo, Anthony P, Chen, Zhi-Ying, Kay, Mark A, Naldini, Luigi, Freeman, Gordon, Tolar, Jakub, Blazar, Bruce R
Publicado em 2011

Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology

Por Osborn, Mark J., Webber, Beau R., McElmurry, Ronald T., Rudser, Kyle D., DeFeo, Anthony P., Muradian, Michael, Petryk, Anna, Hallgrimsson, Benedikt, Blazar, Bruce R., Tolar, Jakub, Braunlin, Elizabeth A.
Publicado no J Inherit Metab Dis (2016)

CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia

Por Osborn, Mark J., Lonetree, Cara-lin, Webber, Beau R., Patel, Dharmeshkumar, Dunmire, Samantha, DeFeo, Anthony P., McElroy, Amber N., MacMillan, Margaret L., Wagner, John E., Blazar, Bruce R., Tolar, Jakub
Publicado no Stem Cells Dev (2016)

Fanconi Anemia Gene Editing by the CRISPR/Cas9 System

Por Osborn, Mark J., Gabriel, Richard, Webber, Beau R., DeFeo, Anthony P., McElroy, Amber N., Jarjour, Jordan, Starker, Colby G., Wagner, John E., Joung, J. Keith, Voytas, Daniel F., von Kalle, Christof, Schmidt, Manfred, Blazar, Bruce R., Tolar, Jakub
Publicado no Hum Gene Ther (2015)

CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa

Por Webber, Beau R, Osborn, Mark J, McElroy, Amber N, Twaroski, Kirk, Lonetree, Cara-lin, DeFeo, Anthony P, Xia, Lily, Eide, Cindy, Lees, Christopher J, McElmurry, Ron T, Riddle, Megan J, Kim, Chong Jai, Patel, Dharmeshkumar D, Blazar, Bruce R, Tolar, Jakub
Publicado no NPJ Regen Med (2016)

TALEN-based Gene Correction for Epidermolysis Bullosa

Por Osborn, Mark J, Starker, Colby G, McElroy, Amber N, Webber, Beau R, Riddle, Megan J, Xia, Lily, DeFeo, Anthony P, Gabriel, Richard, Schmidt, Manfred, Von Kalle, Christof, Carlson, Daniel F, Maeder, Morgan L, Joung, J Keith, Wagner, John E, Voytas, Daniel F, Blazar, Bruce R, Tolar, Jakub
Publicado em 2013

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases

Por Osborn, Mark J, Webber, Beau R, Knipping, Friederike, Lonetree, Cara-lin, Tennis, Nicole, DeFeo, Anthony P, McElroy, Amber N, Starker, Colby G, Lee, Catherine, Merkel, Sarah, Lund, Troy C, Kelly-Spratt, Karen S, Jensen, Michael C, Voytas, Daniel F, von Kalle, Christof, Schmidt, Manfred, Gabriel, Richard, Hippen, Keli L, Miller, Jeffrey S, Scharenberg, Andrew M, Tolar, Jakub, Blazar, Bruce R
Publicado no Mol Ther (2016)