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High density SNP array and reanalysis of genome sequencing uncovers CNVs associated with neurodevelopmental disorders in KOLF2.1J iPSCs.
Gracia-Diaz C, Perdomo JE, Khan ME, Disanza B, Cajka GG, Lei S, Gagne A, Maguire JA, Roule T, Shalem O, Bhoj EJ, Ahrens-Nicklas RC, French D, Goldberg EM, Wang K, Glessner J, Akizu N. Gracia-Diaz C, et al. bioRxiv [Preprint]. 2023 Jun 26:2023.06.26.546614. doi: 10.1101/2023.06.26.546614. bioRxiv. 2023. Update in: Cell Stem Cell. 2024 Mar 7;31(3):288-289. doi: 10.1016/j.stem.2024.02.007 PMID: 37425875 Free PMC article. Updated. Preprint.
Improved specificity and efficacy of base-editing therapies with hybrid guide RNAs.
Whittaker MN, Brooks DL, Quigley A, Jindal I, Said H, Qu P, Wang JZ, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Peranteau WH, Wang X. Whittaker MN, et al. Among authors: ahrens nicklas rc. bioRxiv [Preprint]. 2024 Jun 16:2024.04.22.590531. doi: 10.1101/2024.04.22.590531. bioRxiv. 2024. PMID: 38712058 Free PMC article. Preprint.
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations.
Tricoli L, Sase S, Hacker J, Pham V, Smith S, Chappell M, Breda L, Hurwitz S, Tanaka N, Castracani CC, Guerra A, Hou Z, Schlotawa L, Radhakrishnan K, Kurre P, Ahrens-Nicklas R, Adang L, Vanderver A, Rivella S. Tricoli L, et al. bioRxiv [Preprint]. 2024 Mar 14:2024.03.14.584404. doi: 10.1101/2024.03.14.584404. bioRxiv. 2024. PMID: 38559013 Free PMC article. Preprint.
Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.
Pham V, Tricoli L, Hong X, Wongkittichote P, Castruccio Castracani C, Guerra A, Schlotawa L, Adang LA, Kuhs A, Cassidy MM, Kane O, Tsai E, Presa M, Lutz C, Rivella SB, Ahrens-Nicklas RC. Pham V, et al. Among authors: ahrens nicklas rc. Mol Ther. 2024 Nov 6;32(11):3829-3846. doi: 10.1016/j.ymthe.2024.08.015. Epub 2024 Aug 22. Mol Ther. 2024. PMID: 39169621
Utility of genome sequencing in exome-negative pediatric patients with neurodevelopmental phenotypes.
Nomakuchi TT, Teferedegn EY, Li D, Muirhead KJ, Dubbs H, Leonard J, Muraresku C, Sergio E, Arnold K, Pizzino A, Skraban CM, Zackai EH, Wang K, Ganetzky RD, Vanderver AL, Ahrens-Nicklas RC, Bhoj EJK. Nomakuchi TT, et al. Among authors: ahrens nicklas rc. Am J Med Genet A. 2024 Dec;194(12):e63817. doi: 10.1002/ajmg.a.63817. Epub 2024 Jul 19. Am J Med Genet A. 2024. PMID: 39031459
Progesterone for Neurodevelopment in Fetuses With Congenital Heart Defects: A Randomized Clinical Trial.
Gaynor JW, Moldenhauer JS, Zullo EE, Burnham NB, Gerdes M, Bernbaum JC, D'Agostino JA, Linn RL, Klepczynski B, Randazzo I, Gionet G, Choi GH, Karaj A, Russell WW, Zackai EH, Johnson MP, Gebb JS, Soni S, DeBari SE, Szwast AL, Ahrens-Nicklas RC, Drivas TG, Jacobwitz M, Licht DJ, Vossough A, Nicolson SC, Spray TL, Rychik J, Putt ME. Gaynor JW, et al. Among authors: ahrens nicklas rc. JAMA Netw Open. 2024 May 1;7(5):e2412291. doi: 10.1001/jamanetworkopen.2024.12291. JAMA Netw Open. 2024. PMID: 38805228 Free PMC article. Clinical Trial.
Lmod2 is necessary for effective skeletal muscle contraction.
Larrinaga TM, Farman GP, Mayfield RM, Yuen M, Ahrens-Nicklas RC, Cooper ST, Pappas CT, Gregorio CC. Larrinaga TM, et al. Among authors: ahrens nicklas rc. Sci Adv. 2024 Mar 15;10(11):eadk1890. doi: 10.1126/sciadv.adk1890. Epub 2024 Mar 13. Sci Adv. 2024. PMID: 38478604 Free PMC article.
58 results