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Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington's disease.
Zeitler B, Froelich S, Marlen K, Shivak DA, Yu Q, Li D, Pearl JR, Miller JC, Zhang L, Paschon DE, Hinkley SJ, Ankoudinova I, Lam S, Guschin D, Kopan L, Cherone JM, Nguyen HB, Qiao G, Ataei Y, Mendel MC, Amora R, Surosky R, Laganiere J, Vu BJ, Narayanan A, Sedaghat Y, Tillack K, Thiede C, Gärtner A, Kwak S, Bard J, Mrzljak L, Park L, Heikkinen T, Lehtimäki KK, Svedberg MM, Häggkvist J, Tari L, Tóth M, Varrone A, Halldin C, Kudwa AE, Ramboz S, Day M, Kondapalli J, Surmeier DJ, Urnov FD, Gregory PD, Rebar EJ, Muñoz-Sanjuán I, Zhang HS. Zeitler B, et al. Among authors: froelich s. Nat Med. 2019 Jul;25(7):1131-1142. doi: 10.1038/s41591-019-0478-3. Epub 2019 Jul 1. Nat Med. 2019. PMID: 31263285
Identifying therapeutic drug targets using bidirectional effect genes.
Estrada K, Froelich S, Wuster A, Bauer CR, Sterling T, Clark WT, Ru Y, Trinidad M, Nguyen HP, Luu AR, Wendt DJ, Yogalingam G, Yu GK, LeBowitz JH, Cardon LR. Estrada K, et al. Among authors: froelich s. Nat Commun. 2021 Apr 13;12(1):2224. doi: 10.1038/s41467-021-21843-8. Nat Commun. 2021. PMID: 33850126 Free PMC article.
Lentiviral vectors for immune cells targeting.
Froelich S, Tai A, Wang P. Froelich S, et al. Immunopharmacol Immunotoxicol. 2010 Jun;32(2):208-18. doi: 10.3109/08923970903420582. Immunopharmacol Immunotoxicol. 2010. PMID: 20085508 Free PMC article. Review.
Haploinsufficiency underlies the neurodevelopmental consequences of SLC6A1 variants.
Silva DB, Trinidad M, Ljungdahl A, Revalde JL, Berguig GY, Wallace W, Patrick CS, Bomba L, Arkin M, Dong S, Estrada K, Hutchinson K, LeBowitz JH, Schlessinger A, Johannesen KM, Møller RS, Giacomini KM, Froelich S, Sanders SJ, Wuster A. Silva DB, et al. Among authors: froelich s. Am J Hum Genet. 2024 Jun 6;111(6):1222-1238. doi: 10.1016/j.ajhg.2024.04.021. Epub 2024 May 22. Am J Hum Genet. 2024. PMID: 38781976 Free PMC article.
219 results