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Targeting neuronal function for CNS drug discovery.
Hempel CM, Werley CA, Dempsey GT, Gerber DJ. Hempel CM, et al. Among authors: dempsey gt. Drug Discov Today Technol. 2017 Mar;23:17-25. doi: 10.1016/j.ddtec.2017.03.005. Epub 2017 May 6. Drug Discov Today Technol. 2017. PMID: 28647082 Review.
Homozygous might be hemizygous: CRISPR/Cas9 editing in iPSCs results in detrimental on-target defects that escape standard quality controls.
Simkin D, Papakis V, Bustos BI, Ambrosi CM, Ryan SJ, Baru V, Williams LA, Dempsey GT, McManus OB, Landers JE, Lubbe SJ, George AL Jr, Kiskinis E. Simkin D, et al. Among authors: dempsey gt. Stem Cell Reports. 2022 Apr 12;17(4):993-1008. doi: 10.1016/j.stemcr.2022.02.008. Epub 2022 Mar 10. Stem Cell Reports. 2022. PMID: 35276091 Free PMC article.
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models.
Williams LA, Gerber DJ, Elder A, Tseng WC, Baru V, Delaney-Busch N, Ambrosi C, Mahimkar G, Joshi V, Shah H, Harikrishnan K, Upadhyay H, Rajendran SH, Dhandapani A, Meier J, Ryan SJ, Lewarch C, Black L, Douville J, Cinquino S, Legakis H, Nalbach K, Behrends C, Sato A, Galluzzi L, Yu TW, Brown D, Agrawal S, Margulies D, Kopin A, Dempsey GT. Williams LA, et al. Among authors: dempsey gt. Mol Ther Nucleic Acids. 2022 Jun 22;29:189-203. doi: 10.1016/j.omtn.2022.06.015. eCollection 2022 Sep 13. Mol Ther Nucleic Acids. 2022. PMID: 35860385 Free PMC article.
24 results