Gene editing toward the use of autologous therapies in recessive dystrophic epidermolysis bullosa

Por Perdoni, Christopher, Osborn, Mark J., Tolar, Jakub
Publicado no Transl Res (2015)

Synthetic Zinc Finger Nuclease Design and Rapid Assembly

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

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

Por Osborn, Mark J, McElmurry, Ron T, Peacock, Brandon, Tolar, Jakub, Blazar, Bruce R
Publicado em 2008

Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification

Por Knipping, Friederike, Osborn, Mark J., Petri, Karl, Tolar, Jakub, Glimm, Hanno, von Kalle, Christof, Schmidt, Manfred, Gabriel, Richard
Publicado no Mol Ther Methods Clin Dev (2017)

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

Targeting G with TAL Effectors: A Comparison of Activities of TALENs Constructed with NN and NK Repeat Variable Di-Residues

Por Christian, Michelle L., Demorest, Zachary L., Starker, Colby G., Osborn, Mark J., Nyquist, Michael D., Zhang, Yong, Carlson, Daniel F., Bradley, Philip, Bogdanove, Adam J., Voytas, Daniel F.
Publicado em 2012

Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa

Por Wagner, John E., Ishida-Yamamoto, Akemi, McGrath, John A., Hordinsky, Maria, Keene, Douglas R., Riddle, Megan J., Osborn, Mark J., Lund, Troy, Dolan, Michelle, Blazar, Bruce R., Tolar, Jakub
Publicado em 2010

Keratinocytes from Induced Pluripotent Stem Cells in Junctional Epidermolysis Bullosa

Por Tolar, Jakub, Xia, Lily, Lees, Chris J, Riddle, Megan, McElroy, Amber, Keene, Douglas R, Lund, Troy C, Osborn, Mark J, Marinkovich, M. Peter, Blazar, Bruce R, Wagner, John E
Publicado em 2012

Induced Pluripotent Stem Cells from Individuals with Recessive Dystrophic Epidermolysis Bullosa

Por Tolar, Jakub, Xia, Lily, Riddle, Megan, Lees, Chris J., Eide, Cindy, McElmurry, Ron T., Titeux, Matthias, Osborn, Mark J., Lund, Troy C., Hovnanian, Alain, Wagner, John E., Blazar, Bruce R.
Publicado em 2010

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)

Short-term inhibition of p53 combined with keratinocyte growth factor improves thymic epithelial cell recovery and enhances T-cell reconstitution after murine bone marrow transplan...

Por Kelly, Ryan M., Goren, Emily M., Taylor, Patricia A., Mueller, Scott N., Stefanski, Heather E., Osborn, Mark J., Scott, Hamish S., Komarova, Elena A., Gudkov, Andrei V., Holländer, Georg A., Blazar, Bruce R.
Publicado em 2010

Patient-specific naturally gene-reverted induced pluripotent stem cells in recessive dystrophic epidermolysis bullosa

Por Tolar, Jakub, McGrath, John A., Xia, Lily, Riddle, Megan, Lees, Chris J., Eide, Cindy, Keene, Douglas R., Liu, Lu, Osborn, Mark J., Lund, Troy C., Blazar, Bruce R., Wagner, John E.
Publicado em 2013

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)

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

Por Tolar, Jakub, Park, In-Hyun, Xia, Lily, Lees, Chris J., Peacock, Brandon, Webber, Beau, McElmurry, Ron T., Eide, Cindy R., Orchard, Paul J., Kyba, Michael, Osborn, Mark J., Lund, Troy C., Wagner, John E., Daley, George Q., Blazar, Bruce R.
Publicado em 2011

Minicircle DNA is Superior to Plasmid DNA in Eliciting Antigen-specific CD8(+) T-cell Responses

Por Dietz, Wynette M, Skinner, Nicole E B, Hamilton, Sara E, Jund, Michelle D, Heitfeld, Suzanne M, Litterman, Adam J, Hwu, Patrick, Chen, Zhi-Ying, Salazar, Andres M, Ohlfest, John R, Blazar, Bruce R, Pennell, Christopher A, Osborn, Mark J
Publicado em 2013

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

B7-H3 expression in donor T cells and host cells negatively regulates acute graft-versus-host disease lethality

Por Veenstra, Rachelle G., Flynn, Ryan, Kreymborg, Katharina, McDonald-Hyman, Cameron, Saha, Asim, Taylor, Patricia A., Osborn, Mark J., Panoskaltsis-Mortari, Angela, Schmitt-Graeff, Annette, Lieberknect, Elisabeth, Murphy, William J., Serody, Jonathan S., Munn, David H., Freeman, Gordon J., Allison, James P., Mak, Tak W., van den Brink, Marcel, Zeiser, Robert, Blazar, Bruce R.
Publicado no Blood (2015)

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)