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Perspectives on best practices for gene therapy programs.
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK. Cheever TR, et al. Among authors: chamberlain js. Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3. Hum Gene Ther. 2015. PMID: 25654329 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. 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.
Split intein-mediated protein trans-splicing to express large dystrophins.
Tasfaout H, Halbert CL, McMillen TS, Allen JM, Reyes TR, Flint GV, Grimm D, Hauschka SD, Regnier M, Chamberlain JS. Tasfaout H, et al. Among authors: chamberlain js. Nature. 2024 Aug;632(8023):192-200. doi: 10.1038/s41586-024-07710-8. Epub 2024 Jul 17. Nature. 2024. PMID: 39020181 Free PMC article.
In vivo selection of synthetic nucleocapsids for tissue targeting.
Olshefsky A, Benasutti H, Sylvestre M, Butterfield GL, Rocklin GJ, Richardson C, Hicks DR, Lajoie MJ, Song K, Leaf E, Treichel C, Decarreau J, Ke S, Kher G, Carter L, Chamberlain JS, Baker D, King NP, Pun SH. Olshefsky A, et al. Among authors: chamberlain js. Proc Natl Acad Sci U S A. 2023 Nov 14;120(46):e2306129120. doi: 10.1073/pnas.2306129120. Epub 2023 Nov 8. Proc Natl Acad Sci U S A. 2023. PMID: 37939083 Free PMC article.
251 results