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Molecular architecture determines brain delivery of a transferrin receptor-targeted lysosomal enzyme.
Arguello A, Mahon CS, Calvert MEK, Chan D, Dugas JC, Pizzo ME, Thomsen ER, Chau R, Damo LA, Duque J, Fang M, Giese T, Kim DJ, Liang N, Nguyen HN, Solanoy H, Tsogtbaatar B, Ullman JC, Wang J, Dennis MS, Diaz D, Gunasekaran K, Henne KR, Lewcock JW, Sanchez PE, Troyer MD, Harris JM, Scearce-Levie K, Shan L, Watts RJ, Thorne RG, Henry AG, Kariolis MS. Arguello A, et al. Among authors: ullman jc. J Exp Med. 2022 Mar 7;219(3):e20211057. doi: 10.1084/jem.20211057. Epub 2022 Feb 28. J Exp Med. 2022. PMID: 35226042 Free PMC article.
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice.
Ullman JC, Arguello A, Getz JA, Bhalla A, Mahon CS, Wang J, Giese T, Bedard C, Kim DJ, Blumenfeld JR, Liang N, Ravi R, Nugent AA, Davis SS, Ha C, Duque J, Tran HL, Wells RC, Lianoglou S, Daryani VM, Kwan W, Solanoy H, Nguyen H, Earr T, Dugas JC, Tuck MD, Harvey JL, Reyzer ML, Caprioli RM, Hall S, Poda S, Sanchez PE, Dennis MS, Gunasekaran K, Srivastava A, Sandmann T, Henne KR, Thorne RG, Di Paolo G, Astarita G, Diaz D, Silverman AP, Watts RJ, Sweeney ZK, Kariolis MS, Henry AG. Ullman JC, et al. Sci Transl Med. 2020 May 27;12(545):eaay1163. doi: 10.1126/scitranslmed.aay1163. Sci Transl Med. 2020. PMID: 32461331
Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders.
Ullman JC, Mellem KT, Xi Y, Ramanan V, Merritt H, Choy R, Gujral T, Young LEA, Blake K, Tep S, Homburger JR, O'Regan A, Ganesh S, Wong P, Satterfield TF, Lin B, Situ E, Yu C, Espanol B, Sarwaikar R, Fastman N, Tzitzilonis C, Lee P, Reiton D, Morton V, Santiago P, Won W, Powers H, Cummings BB, Hoek M, Graham RR, Chandriani SJ, Bainer R, DePaoli-Roach AA, Roach PJ, Hurley TD, Sun RC, Gentry MS, Sinz C, Dick RA, Noonberg SB, Beattie DT, Morgans DJ Jr, Green EM. Ullman JC, et al. Sci Transl Med. 2024 Jan 17;16(730):eadf1691. doi: 10.1126/scitranslmed.adf1691. Epub 2024 Jan 17. Sci Transl Med. 2024. PMID: 38232139
The structural mechanism of human glycogen synthesis by the GYS1-GYG1 complex.
Fastman NM, Liu Y, Ramanan V, Merritt H, Ambing E, DePaoli-Roach AA, Roach PJ, Hurley TD, Mellem KT, Ullman JC, Green E, Morgans D Jr, Tzitzilonis C. Fastman NM, et al. Among authors: ullman jc. Cell Rep. 2022 Jul 5;40(1):111041. doi: 10.1016/j.celrep.2022.111041. Cell Rep. 2022. PMID: 35793618 Free article.
High-Throughput Liquid Chromatography-Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II.
Wang J, Bhalla A, Ullman JC, Fang M, Ravi R, Arguello A, Thomsen E, Tsogtbaatar B, Guo JL, Skuja LL, Dugas JC, Davis SS, Poda SB, Gunasekaran K, Costanzo S, Sweeney ZK, Henry AG, Harris JM, Henne KR, Astarita G. Wang J, et al. Among authors: ullman jc. Int J Mol Sci. 2020 Jul 30;21(15):5449. doi: 10.3390/ijms21155449. Int J Mol Sci. 2020. PMID: 32751752 Free PMC article.