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A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.
Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S, Camarena J, Lee CM, Park SH, Wiebking V, Bak RO, Gomez-Ospina N, Pavel-Dinu M, Sun W, Bao G, Porteus MH, Behlke MA. Vakulskas CA, et al. Nat Med. 2018 Aug;24(8):1216-1224. doi: 10.1038/s41591-018-0137-0. Epub 2018 Aug 6. Nat Med. 2018. PMID: 30082871 Free PMC article.
Identification of preexisting adaptive immunity to Cas9 proteins in humans.
Charlesworth CT, Deshpande PS, Dever DP, Camarena J, Lemgart VT, Cromer MK, Vakulskas CA, Collingwood MA, Zhang L, Bode NM, Behlke MA, Dejene B, Cieniewicz B, Romano R, Lesch BJ, Gomez-Ospina N, Mantri S, Pavel-Dinu M, Weinberg KI, Porteus MH. Charlesworth CT, et al. Among authors: Vakulskas CA. Nat Med. 2019 Feb;25(2):249-254. doi: 10.1038/s41591-018-0326-x. Epub 2019 Jan 28. Nat Med. 2019. PMID: 30692695 Free PMC article.
Evaluation and Reduction of CRISPR Off-Target Cleavage Events.
Vakulskas CA, Behlke MA. Vakulskas CA, et al. Nucleic Acid Ther. 2019 Aug;29(4):167-174. doi: 10.1089/nat.2019.0790. Epub 2019 May 20. Nucleic Acid Ther. 2019. PMID: 31107154 Free PMC article.
Non-nucleotide Modification of Anti-miRNA Oligonucleotides.
Lennox KA, Vakulskas CA, Behlke MA. Lennox KA, et al. Among authors: Vakulskas CA. Methods Mol Biol. 2017;1517:51-69. doi: 10.1007/978-1-4939-6563-2_3. Methods Mol Biol. 2017. PMID: 27924473
Circuitry linking the Csr and stringent response global regulatory systems.
Edwards AN, Patterson-Fortin LM, Vakulskas CA, Mercante JW, Potrykus K, Vinella D, Camacho MI, Fields JA, Thompson SA, Georgellis D, Cashel M, Babitzke P, Romeo T. Edwards AN, et al. Among authors: Vakulskas CA. Mol Microbiol. 2011 Jun;80(6):1561-80. doi: 10.1111/j.1365-2958.2011.07663.x. Epub 2011 May 5. Mol Microbiol. 2011. PMID: 21488981 Free PMC article.
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