Publications
Photo by Greg Dunn
2020 - Present:
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Phuong HT, Tomas RF, Akmese C, Mijares A, Gerstin IM, Guo S, Bell LR, Ellwood R, Yegorova S, Ng SK, Massey G, Phillips J, Melloni A, Pletnikova O, Lou X, Clark HB, Troncoso JC, Hyman BT, Prokop S, Ranum LP, Nguyen L. PolyGR-containing aggregates link with pathology and clinical features of Alzheimer's disease. Acta Neuropathol. 2025 Nov 8;150(1):49.
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Association of polyGR+ aggregate levels in AD hippocampus with known disease
neuropathological hallmarks​
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​​Nguyen, L.*; Ajredini, R.; Guo, S.; Lisa E. L. Romano, L. E. L; Tomas, R. F.; Logan, B. R.; Ranum, P.; Zu, T.; Coronel, M. B.; Kelley, C. P.; Redding-Ochoa, J.; , Nizamis, E.;, Melloni, A.; Connors, T. R.; Gaona, A.; Thangaraju, K.;, Pletnikova, O.;, Clark, H. B., Davidson, B. L.; Yachnis, T.; Golde, T.; Lou, X. Y.;13, Wang, E. T.; Renton, A. E.; Goate, A.; Valdmanis, P. N.; Prokop, S.; Troncoso, J. C.;, Hyman, B.; Ranum, L. P. W.* CASP8 intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer’s disease risk, PNAS, 2025, 122(7) :e2416885122. PMID: 39937857, * co-corresponding author.
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Highlighted on the CNG website , the UF Health, Alzforum
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dCas9READ for pulling down & identifying novel repeat expansions (A) and pathology-to-gene identification strategy for detecting disease-associated repeat expansion mutations (B)
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Nguyen, L. Updates on Disease Mechanisms and Therapeutics for Amyotrophic Lateral Sclerosis, Cells. 2024, 13(11):888. PMID: 38891021 ​
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Nguyen, L. RNA therapeutics for neurological disease, Prog. Mol. Biol. Transl. Sci. 2024, 203, 165-180. PMID: 38359997​
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Guo, S.; Nguyen, L*; Ranum, L. P. W*. RAN proteins in neurodegenerative disease: repeating themes and unifying therapeutic strategies, Curr. Opin. Neurobiol. 2022, 72, 160-170. PMID: 34953315 (*co-corresponding author)
Tusi, S. K.; Nguyen, L.; Thangaraju, K.; Cleary, J. D.; Zu, T.; Ranum, L. P. W. The alternative initiation factor eIF2A plays key role in RAN translation of myotonic dystrophy type 2 CCUG• CAGG repeats, Hum. Mol. Genet. 2021, 30, 1020-1029. PMID: 33856033
Pattamatta. A.; Nguyen, L.; Olafson, H.; Scotti, M.; Liu, Y.; Richardson, J.; Berglund, A. J.; Zu, T.; Wang E. T.; Ranum, L. P. W. Repeat length increases disease penetrance and severity in C9orf72 ALS/FTD BAC transgenic mice, Hum. Mol. Genet. 2021, 29, 3900-3918. PMID: 33378537
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Nguyen, L.*; Laboissonniere, L. A.*; Guo, S.*; Pilotto, F.; Scheidegger, O.; Oestmann, A.; Hammond J. W.; Li, H.; Hyysalo, A.; Peltola, R.; Pattamatta, A.; Zu, T.; Voutilainen, M. H.; Gelbard, H. A.; Saxena, S.; Ranum, L. P. W. Survival and motor phenotypes in FVB C9-500 ALS/FTD BAC transgenic mice reproduced by multiple labs, Neuron, 2020, 108, 1-13. (*co-first author). PMID: 33022226
Zu, T.; Bardhi, O.; Guo. S.; Ryskamp, D.; Tusi S. K; Liu, Y.; Klippel, K.; Chakrabarty, P.; Nguyen, L.; Golde T. E., Sonenberg, N.; Ranum L. P. W. Metformin inhibits RAN translation through PKR pathway and corrects ALS/FTD phenotypes in C9orf72 mouse model. Proc. Natl. Acad. Sci. U.S.A, 2020, 117(31), 18591-18599. PMID: 32690681
Nguyen, L.; Montrasio, M.; Pattamatta, A.; Tusi, S. K.; Bardhi, O.; Meyer, K. D.; Hayes, L.; Nakamura, K.; Banez-Coronel, M.; Coyne, A.; Guo, S.; Laboissonniere, L. A.; Gu, Y.; Narayanan, S.; Smith, B.; Nitsch, R. M.; Kankel, M. W.; Rushe, M.; Rothstein, J.; Zu, T.; Grimm, J.; Ranum, L. P. W. Antibody therapy targeting RAN proteins rescues C9 ALS/FTD in C9orf72 mouse model, Neuron, 2020, 105(4), 645-662. PMID: 31831332
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2015-2019:
Nguyen, L.; Cleary, J. D.; Ranum, L. P. W. Repeat associated non-ATG translation: molecular mechanisms and contribution to neurologic disease, Annu. Rev. Neurosci. 2019, 42, 227-24. PMID: 30909783
Chu D. T.; Minh Nguyet N. T.; Nga V. T.; Thai Lien N. V.; Vo D. D.; Nguyen L.; Nhu Ngoc V. T.; Son L. H.; Le D. H.; Nga V. B.; Van Tu P.; Van To T.; Ha L. S.; Tao Y.; Pham V. H.; An update on obesity: Mental consequences and psychological interventions, Diabetes Metab Syndr. 2019; 13(1), 155-160. PMID: 30641689
Bai, Y.*; Nguyen, L.*; Song, Z.*; Peng, S.; Zheng, N.; Lee, J.; Kapoor, I.; Cheng, J.; Chan, W.-Y.; Zimmerman, S. C. Integrating Display and Delivery Functionality with a Cell Penetrating Peptide Mimic as a Scaffold for Intracellular Multivalent Multi-targeting, J. Am. Chem. Soc. 2016, 138, 9498–9507. (*co-first author). PMID: 27355522
Luu, M. L*; Nguyen, L.*; Chan, W.-Y.; Zimmerman, S. C. A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy, ChemMedChem 2016, 11, 1428-1435. (*co-first author). PMID: 27245480
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Nguyen, L.; Luu, M. L.; Peng, S.; Serrano, F. J; Chan, W.-Y.; Zimmerman, S. C. Rationally designed small molecules that target both the DNA and RNA causing myotonic dystrophy type 1, J. Am. Chem. Soc. 2015, 14180–14189. (UIUC news). PMID: 26473464
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Before 2015:
Nguyen, L.; Lee, J.; Wong, C.-H.; Zimmerman, S. C. Small Molecules that Target the Toxic RNA in Myotonic Dystrophy Type 2, ChemMedChem 2014, 9, 2455–2462. (designated VIP, highlighted on the journal cover). PMID: 24938413
Wong, C.-H.; Nguyen, L.; Peh, J.; Luu, L. M.; Sanchez, J. S.; Richardson, S. L.; Tuccinardi, T.; Ho, T.; Chan, E. H. Y.; Chan, W.-Y.; Baranger, A. M.; Hergenrother, P. J.; Zimmerman, S. C. Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor, J. Am. Chem. Soc. 2014, 136, 6355−6361. PMID: 24702247
Jahromi, A. H.; Fu, Y.*; Miller, K. A.*; Nguyen, L.*; Luu M. L.; Baranger, A. M.; Zimmerman, S. C. Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1, J. Med. Chem. 2013, 56, 9471–9481. (*co-second author). PMID: 24188018
Jahromi, A. H.; Nguyen, L.; Fu, Y.; Miller, K. A.; Baranger, A. M.; Zimmerman, S. C. A novel CUGexp·MBNL1 inhibitor with therapeutic potential for myotonic dystrophy type 1, ACS Chem. Biol. 2013, 8, 1037–1043. (highlighted on the journal cover and UIUC news). PMID: 23480597
Do, Q. H.; Pham V. T.; Nguyen, L.; Do, Q. C. Analysis of compounds in Aloe vera by gas chromatography-mass spectrometry, Science and Technology office, Vietnam 2010, 2.
