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2010 1
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2020 2
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Page 1
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Birch SM, Lawlor MW, Conlon TJ, Guo LJ, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, Brown KJ, Golebiowski D, Bettis AK, Balog-Alvarez CJ, Clement N, Coleman KE, Corti M, Pan X, Hauschka SD, Gonzalez JP, Morris CA, Schneider JS, Duan D, Chamberlain JS, Byrne BJ, Kornegay JN. Birch SM, et al. Among authors: golebiowski d. Sci Transl Med. 2023 Jan 4;15(677):eabo1815. doi: 10.1126/scitranslmed.abo1815. Epub 2023 Jan 4. Sci Transl Med. 2023. PMID: 36599002
A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes.
van Lieshout LP, Rubin M, Costa-Grant K, Ota S, Golebiowski D, Panico T, Wiberg E, Szymczak K, Gilmore R, Stanvick M, Burnham B, Gagnon J, Iwuchukwu I, Yang G, Ghazi I, Meola A, Dickerson R, Thiers T, Mustich L, Hayes A, Rivas I, Lotterhand J, Avila N, McGivney J, Yin J, Kelly T. van Lieshout LP, et al. Among authors: golebiowski d. Mol Ther Methods Clin Dev. 2023 May 8;29:426-436. doi: 10.1016/j.omtm.2023.05.004. eCollection 2023 Jun 8. Mol Ther Methods Clin Dev. 2023. PMID: 37273900 Free PMC article.
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs.
Osborn MF, Coles AH, Golebiowski D, Echeverria D, Moazami MP, Watts JK, Sena-Esteves M, Khvorova A. Osborn MF, et al. Among authors: golebiowski d. Mol Cancer Ther. 2018 Jun;17(6):1251-1258. doi: 10.1158/1535-7163.MCT-17-1144. Epub 2018 Apr 13. Mol Cancer Ther. 2018. PMID: 29654062 Free PMC article.
Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/mdx Mice.
Kennedy TL, Guiraud S, Edwards B, Squire S, Moir L, Babbs A, Odom G, Golebiowski D, Schneider J, Chamberlain JS, Davies KE. Kennedy TL, et al. Among authors: golebiowski d. Mol Ther Methods Clin Dev. 2018 Oct 16;11:92-105. doi: 10.1016/j.omtm.2018.10.005. eCollection 2018 Dec 14. Mol Ther Methods Clin Dev. 2018. PMID: 30417024 Free PMC article.
A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy.
Wong TWY, Ahmed A, Yang G, Maino E, Steiman S, Hyatt E, Chan P, Lindsay K, Wong N, Golebiowski D, Schneider J, Delgado-Olguín P, Ivakine EA, Cohn RD. Wong TWY, et al. Among authors: golebiowski d. Dis Model Mech. 2020 Sep 21;13(9):dmm045369. doi: 10.1242/dmm.045369. Dis Model Mech. 2020. PMID: 32988972 Free PMC article.
Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain.
Golebiowski D, van der Bom IMJ, Kwon CS, Miller AD, Petrosky K, Bradbury AM, Maitland S, Kühn AL, Bishop N, Curran E, Silva N, GuhaSarkar D, Westmoreland SV, Martin DR, Gounis MJ, Asaad WF, Sena-Esteves M. Golebiowski D, et al. Hum Gene Ther. 2017 Jun;28(6):510-522. doi: 10.1089/hum.2016.109. Epub 2017 Jan 26. Hum Gene Ther. 2017. PMID: 28132521 Free PMC article.
12 results