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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
Davidson BL, Gao G, Berry-Kravis E, Bradbury AM, Bönnemann C, Buxbaum JD, Corcoran GR, Gray SJ, Gray-Edwards H, Kleiman RJ, Shaywitz AJ, Wang D, Zoghbi HY, Flotte TR, Tauscher-Wisniewski S, Tifft CJ, Sahin M; Gene Therapy Workshop Faculty. Davidson BL, et al. Among authors: gao g. Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17. Mol Ther. 2022. PMID: 35585789 Free PMC article. Review.
Long-term, efficient inhibition of microRNA function in mice using rAAV vectors.
Xie J, Ameres SL, Friedline R, Hung JH, Zhang Y, Xie Q, Zhong L, Su Q, He R, Li M, Li H, Mu X, Zhang H, Broderick JA, Kim JK, Weng Z, Flotte TR, Zamore PD, Gao G. Xie J, et al. Among authors: gao g. Nat Methods. 2012 Mar 4;9(4):403-9. doi: 10.1038/nmeth.1903. Nat Methods. 2012. PMID: 22388288 Free PMC article.
Gene transfer in the lung using recombinant adeno-associated virus.
Gruntman AM, Mueller C, Flotte TR, Gao G. Gruntman AM, et al. Among authors: gao g. Curr Protoc Microbiol. 2012 Aug;Chapter 14:Unit14D.2. doi: 10.1002/9780471729259.mc14d02s26. Curr Protoc Microbiol. 2012. PMID: 22875566 Free PMC article.
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Among authors: gao g. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
6,194 results