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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: williams la. 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. 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.
Deep functional measurements of Fragile X syndrome human neurons reveal multiparametric electrophysiological disease phenotype.
Fink JJ, Delaney-Busch N, Dawes R, Nanou E, Folts C, Harikrishnan K, Hempel C, Upadhyay H, Nguyen T, Shroff H, Stoppel D, Ryan SJ, Jacques J, Grooms J, Berry-Kravis E, Bear MF, Williams LA, Gerber D, Bunnage M, Furey B, Dempsey GT. Fink JJ, et al. Among authors: williams la. Commun Biol. 2024 Nov 6;7(1):1447. doi: 10.1038/s42003-024-07120-6. Commun Biol. 2024. PMID: 39506078 Free PMC article.
How to make spinal motor neurons.
Davis-Dusenbery BN, Williams LA, Klim JR, Eggan K. Davis-Dusenbery BN, et al. Among authors: williams la. Development. 2014 Feb;141(3):491-501. doi: 10.1242/dev.097410. Development. 2014. PMID: 24449832 Review.
551 results