Synthetic Zinc Finger Nuclease Design and Rapid Assembly

af Osborn, Mark J., DeFeo, Anthony P., Blazar, Bruce R., Tolar, Jakub
Udgivet 2011

CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells

af Skvarova Kramarzova, Karolina, Osborn, Mark J., Webber, Beau R., DeFeo, Anthony P., McElroy, Amber N., Kim, Chong Jai, Tolar, Jakub
Udgivet i Int J Mol Sci (2017)

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

af 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
Udgivet 2011

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

af 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.
Udgivet i J Inherit Metab Dis (2016)

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

af 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
Udgivet i Stem Cells Dev (2016)

Fanconi Anemia Gene Editing by the CRISPR/Cas9 System

af 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
Udgivet i Hum Gene Ther (2015)

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

af 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
Udgivet i NPJ Regen Med (2016)

TALEN-based Gene Correction for Epidermolysis Bullosa

af 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
Udgivet 2013

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

af 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
Udgivet i Mol Ther (2016)