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Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Low RLY, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: fang m. Cell. 2021 Sep 2;184(18):4651-4668.e25. doi: 10.1016/j.cell.2021.08.002. Epub 2021 Aug 26. Cell. 2021. PMID: 34450028 Free PMC article.
Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
Bhalla A, Ravi R, Fang M, Arguello A, Davis SS, Chiu CL, Blumenfeld JR, Nguyen HN, Earr TK, Wang J, Astarita G, Zhu Y, Fiore D, Scearce-Levie K, Diaz D, Cahan H, Troyer MD, Harris JM, Escolar ML. Bhalla A, et al. Among authors: fang m. Int J Mol Sci. 2020 Jul 22;21(15):5188. doi: 10.3390/ijms21155188. Int J Mol Sci. 2020. PMID: 32707880 Free PMC 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: fang m. Int J Mol Sci. 2020 Jul 30;21(15):5449. doi: 10.3390/ijms21155449. Int J Mol Sci. 2020. PMID: 32751752 Free PMC article.
Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
Arguello A, Meisner R, Thomsen ER, Nguyen HN, Ravi R, Simms J, Lo I, Speckart J, Holtzman J, Gill TM, Chan D, Cheng Y, Chiu CL, Dugas JC, Fang M, Lopez IA, Solanoy H, Tsogtbaatar B, Zhu Y, Bhalla A, Henne KR, Henry AG, Delucchi A, Costanzo S, Harris JM, Diaz D, Scearce-Levie K, Sanchez PE. Arguello A, et al. Among authors: fang m. JCI Insight. 2021 Oct 8;6(19):e145445. doi: 10.1172/jci.insight.145445. JCI Insight. 2021. PMID: 34622797 Free PMC article.
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: fang m. 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.
Alterations in Lysosomal, Glial and Neurodegenerative Biomarkers in Patients with Sporadic and Genetic Forms of Frontotemporal Dementia.
Hsiao-Nakamoto J, Chiu CL, VandeVrede L, Ravi R, Vandenberg B, De Groot J, Tsogtbaatar B, Fang M, Auger P, Gould NS, Marchioni F, Powers CA, Davis SS, Suh JH, Alkabsh J, Heuer HW, Lago AL, Scearce-Levie K, Seeley WW, Boeve BF, Rosen HJ, Berger A, Tsai R, Di Paolo G, Boxer AL, Bhalla A, Huang F; ALLFTD Consortium. Hsiao-Nakamoto J, et al. Among authors: fang m. bioRxiv [Preprint]. 2024 Feb 12:2024.02.09.579529. doi: 10.1101/2024.02.09.579529. bioRxiv. 2024. PMID: 38405775 Free PMC article. Preprint.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Logan T, Simon MJ, Rana A, Cherf GM, Srivastava A, Davis SS, Yoon Low RL, Chiu CL, Fang M, Huang F, Bhalla A, Llapashtica C, Prorok R, Pizzo ME, Calvert MEK, Sun EW, Hsiao-Nakamoto J, Rajendra Y, Lexa KW, Srivastava DB, van Lengerich B, Wang J, Robles-Colmenares Y, Kim DJ, Duque J, Lenser M, Earr TK, Nguyen H, Chau R, Tsogtbaatar B, Ravi R, Skuja LL, Solanoy H, Rosen HJ, Boeve BF, Boxer AL, Heuer HW, Dennis MS, Kariolis MS, Monroe KM, Przybyla L, Sanchez PE, Meisner R, Diaz D, Henne KR, Watts RJ, Henry AG, Gunasekaran K, Astarita G, Suh JH, Lewcock JW, DeVos SL, Di Paolo G. Logan T, et al. Among authors: fang m. Cell. 2024 Mar 14;187(6):1565-1566. doi: 10.1016/j.cell.2024.02.015. Cell. 2024. PMID: 38490183 No abstract available.
3,970 results