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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: gyorgy b. Sci Adv. 2021 Jan 8;7(2):eabb1703. doi: 10.1126/sciadv.abb1703. Print 2021 Jan. Sci Adv. 2021. PMID: 33523984 Free PMC article.
Exosome-associated AAV vector as a robust and convenient neuroscience tool.
Hudry E, Martin C, Gandhi S, György B, Scheffer DI, Mu D, Merkel SF, Mingozzi F, Fitzpatrick Z, Dimant H, Masek M, Ragan T, Tan S, Brisson AR, Ramirez SH, Hyman BT, Maguire CA. Hudry E, et al. Among authors: gyorgy b. Gene Ther. 2016 Apr;23(4):380-92. doi: 10.1038/gt.2016.11. Epub 2016 Feb 2. Gene Ther. 2016. PMID: 26836117 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. 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.
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. 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.
77 results