Research output per year
Research output per year
Research output
- 11 Article
- 11 results
Search results
-
2023
Cas9-induced targeted integration of large DNA payloads in primary human T cells via homology-mediated end-joining DNA repair
Webber, B. R., Johnson, M. J., Skeate, J. G., Slipek, N. J., Lahr, W. S., DeFeo, A. P., Mills, L. J., Qiu, X., Rathmann, B., Diers, M. D., Wick, B., Henley, T., Choudhry, M., Starr, T. K., McIvor, R. S. & Moriarity, B. S., 2023, (Accepted/In press) In: Nature Biomedical Engineering.Research output: Contribution to journal › Article › peer-review
1 Scopus citations -
2022
Correction of Fanconi Anemia Mutations Using Digital Genome Engineering
Sipe, C. J., Kluesner, M. G., Bingea, S. P., Lahr, W. S., Andrew, A. A., Wang, M., DeFeo, A. P., Hinkel, T. L., Laoharawee, K., Wagner, J. E., MacMillan, M. L., Vercellotti, G. M., Tolar, J., Osborn, M. J., McIvor, R. S., Webber, B. R. & Moriarity, B. S., Aug 2022, In: International journal of molecular sciences. 23, 15, 8416.Research output: Contribution to journal › Article › peer-review
Open Access2 Scopus citations -
2017
Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology
Osborn, M. J., Webber, B. R., McElmurry, R. T., Rudser, K. D., DeFeo, A. P., Muradian, M., Petryk, A., Hallgrimsson, B., Blazar, B. R., Tolar, J. & Braunlin, E. A., Mar 1 2017, In: Journal of Inherited Metabolic Disease. 40, 2, p. 281-289 9 p.Research output: Contribution to journal › Article › peer-review
Open Access11 Scopus citations -
CRISPR/Cas9-mediated correction of the FANCD1 Gene in primary patient cells
Skvarova Kramarzova, K., Osborn, M. J., Webber, B. R., Defeo, A. P., McElroy, A. N., Kim, C. J. & Tolar, J., Jun 14 2017, In: International journal of molecular sciences. 18, 6, 1269.Research output: Contribution to journal › Article › peer-review
Open Access26 Scopus citations -
2016
CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa
Webber, B. R., Osborn, M. J., McElroy, A. N., Twaroski, K., Lonetree, C., Defeo, A. P., Xia, L., Eide, C., Lees, C. J., McElmurry, R. T., Riddle, M. J., Kim, C. J., Patel, D. D., Blazar, B. R. & Tolar, J., Dec 8 2016, In: Regenerative Medicine. 1Research output: Contribution to journal › Article › peer-review
Open Access75 Scopus citations -
CRISPR/Cas9 targeted gene editing and cellular engineering in fanconi anemia
Osborn, M. J., Lonetree, C. L., Webber, B. R., Patel, D. D., Dunmire, S., DeFeo, A. P., McElroy, A. N., MacMillan, M. L., Wagner, J. E., Blazar, B. R. & Tolar, J., Oct 15 2016, In: Stem Cells and Development. 25, 20, p. 1591-1603 13 p.Research output: Contribution to journal › Article › peer-review
Open Access26 Scopus citations -
Evaluation of TCR gene editing achieved by TALENs, CRISPR/Cas9, and megaTAL nucleases
Osborn, M. J., Webber, B. R., Knipping, F., Lonetree, C. L., Tennis, N., DeFeo, A. P., McElroy, A. N., Starker, C. G., Lee, C., Merkel, S., Lund, T. C., Kelly-Spratt, K. S., Jensen, M. C., Voytas, D. F., Von Kalle, C., Schmidt, M., Gabriel, R., Hippen, K. L., Miller, J. S., Scharenberg, A. M., & 2 others, Mar 1 2016, In: Molecular Therapy. 24, 3, p. 570-581 12 p.Research output: Contribution to journal › Article › peer-review
Open Access160 Scopus citations -
2015
Fanconi anemia gene editing by the CRISPR/Cas9 system
Osborn, M. J., Gabriel, R., Webber, B. R., Defeo, A. P., McElroy, A. N., Jarjour, J., Starker, C. G., Wagner, J. E., Joung, J. K., Voytas, D. F., Von Kalle, C., Schmidt, M., Blazar, B. R. & Tolar, J., Feb 1 2015, In: Human gene therapy. 26, 2, p. 114-126 13 p.Research output: Contribution to journal › Article › peer-review
Open Access97 Scopus citations -
2013
TALEN-based gene correction for epidermolysis bullosa
Osborn, M. J., Starker, C. G., McElroy, A. N., Webber, B. R., Riddle, M. J., Xia, L., Defeo, A. P., Gabriel, R., Schmidt, M., Von Kalle, C., Carlson, D. F., Maeder, M. L., Joung, J. K., Wagner, J. E., Voytas, D. F., Blazar, B. R. & Tolar, J., Jun 2013, In: Molecular Therapy. 21, 6, p. 1151-1159 9 p.Research output: Contribution to journal › Article › peer-review
223 Scopus citations -
2011
Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type i
Osborn, M. J., McElmurry, R. T., Lees, C. J., Defeo, A. P., Chen, Z. Y., Kay, M. A., Naldini, L., Freeman, G., Tolar, J. & Blazar, B. R., Mar 2011, In: Molecular Therapy. 19, 3, p. 450-460 11 p.Research output: Contribution to journal › Article › peer-review
81 Scopus citations -
Synthetic zinc finger nuclease design and rapid assembly
Osborn, M. J., Defeo, A. P., Blazar, B. R. & Tolar, J., Sep 1 2011, In: Human gene therapy. 22, 9, p. 1155-1165 11 p.Research output: Contribution to journal › Article › peer-review
Open Access15 Scopus citations