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Mutant Allele-Specific CRISPR Disruption in DYT1 Dystonia Fibroblasts Restores Cell Function.
Cruz L, György B, Cheah PS, Kleinstiver BP, Eimer WA, Garcia SP, Sharma N, Ozelius LJ, Bragg DC, Joung JK, Norberto de Souza O, Macedo Timmers LFS, Breakefield XO. Cruz L, et al. Among authors: breakefield xo. Mol Ther Nucleic Acids. 2020 Sep 4;21:1-12. doi: 10.1016/j.omtn.2020.05.009. Epub 2020 May 15. Mol Ther Nucleic Acids. 2020. PMID: 32502938 Free PMC article.
CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer's Disease.
György B, Lööv C, Zaborowski MP, Takeda S, Kleinstiver BP, Commins C, Kastanenka K, Mu D, Volak A, Giedraitis V, Lannfelt L, Maguire CA, Joung JK, Hyman BT, Breakefield XO, Ingelsson M. György B, et al. Among authors: breakefield xo. Mol Ther Nucleic Acids. 2018 Jun 1;11:429-440. doi: 10.1016/j.omtn.2018.03.007. Epub 2018 Mar 16. Mol Ther Nucleic Acids. 2018. PMID: 29858078 Free PMC article.
Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin.
Cheah PS, Prabhakar S, Yellen D, Beauchamp RL, Zhang X, Kasamatsu S, Bronson RT, Thiele EA, Kwiatkowski DJ, Stemmer-Rachamimov A, György B, Ling KH, Kaneki M, Tannous BA, Ramesh V, Maguire CA, Breakefield XO. Cheah PS, et al. Among authors: breakefield xo. Sci Adv. 2021 Jan 8;7(2):eabb1703. doi: 10.1126/sciadv.abb1703. Print 2021 Jan. Sci Adv. 2021. PMID: 33523984 Free PMC article.
Rescue of Hearing by Gene Delivery to Inner-Ear Hair Cells Using Exosome-Associated AAV.
György B, Sage C, Indzhykulian AA, Scheffer DI, Brisson AR, Tan S, Wu X, Volak A, Mu D, Tamvakologos PI, Li Y, Fitzpatrick Z, Ericsson M, Breakefield XO, Corey DP, Maguire CA. György B, et al. Among authors: breakefield xo. Mol Ther. 2017 Feb 1;25(2):379-391. doi: 10.1016/j.ymthe.2016.12.010. Epub 2017 Jan 9. Mol Ther. 2017. PMID: 28082074 Free PMC article.
High levels of AAV vector integration into CRISPR-induced DNA breaks.
Hanlon KS, Kleinstiver BP, Garcia SP, Zaborowski MP, Volak A, Spirig SE, Muller A, Sousa AA, Tsai SQ, Bengtsson NE, Lööv C, Ingelsson M, Chamberlain JS, Corey DP, Aryee MJ, Joung JK, Breakefield XO, Maguire CA, György B. Hanlon KS, et al. Among authors: breakefield xo. Nat Commun. 2019 Sep 30;10(1):4439. doi: 10.1038/s41467-019-12449-2. Nat Commun. 2019. PMID: 31570731 Free PMC article.
Secretion and Uptake of α-Synuclein Via Extracellular Vesicles in Cultured Cells.
Gustafsson G, Lööv C, Persson E, Lázaro DF, Takeda S, Bergström J, Erlandsson A, Sehlin D, Balaj L, György B, Hallbeck M, Outeiro TF, Breakefield XO, Hyman BT, Ingelsson M. Gustafsson G, et al. Among authors: breakefield xo. Cell Mol Neurobiol. 2018 Nov;38(8):1539-1550. doi: 10.1007/s10571-018-0622-5. Epub 2018 Oct 4. Cell Mol Neurobiol. 2018. PMID: 30288631 Free PMC article.
470 results