Andrew P. Lieberman Lab

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Publications

2022

Species-specific differences in NPC1 protein trafficking govern therapeutic response in Niemann-Pick type C disease
Mark L. Schultz, Kylie J. Schache, Ruth D. Azaria, Esmée Q. Kuiper, Steven Erwood, Evgueni A. Ivakine, Nicole Y. Farhat, Forbes D. Porter, Koralege C. Pathmasiri, Stephanie M. Cologna, Michael D. Uhler, and Andrew P. Lieberman. JCI Insight. 2022 Oct 27;e160308; doi: 10.1172/jci.insight.160308

 

2021

TDP-43 proteinopathy occurs independently of autophagic substrate accumulation and underlies nuclear defects in Niemann-Pick C disease
Liu EA, Mori E, Hamasaki F, Lieberman AP. TDP-43 proteinopathy occurs independently of autophagic substrate accumulation and underlies nuclear defects in Niemann-Pick C disease. Neuropathology and Applied Neurobiology. PMID 34048071 DOI: 10.1111/nan.12738

 

Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage
Ilnytska O, Lai K, Gorshkov K, Schultz ML, Tran BN, Jeziorek M, Kunkel TJ, Azaria RD, McLoughlin HS, Waghalter M, Xu Y, Schlame M, Altan-Bonnet N, Zheng W, Lieberman AP, et al. Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage. The Journal of Biological Chemistry. 100813. PMID 34023384 DOI: 10.1016/j.jbc.2021.100813

 

Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia
Colombo A, Dinkel L, Müller SA, Sebastian Monasor L, Schifferer M, Cantuti-Castelvetri L, König J, Vidatic L, Bremova-Ertl T, Lieberman AP, Hecimovic S, Simons M, Lichtenthaler SF, Strupp M, Schneider SA, et al. Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia. Nature Communications. 12: 1158. PMID 33627648 DOI: 10.1038/s41467-021-21428-5

 

2020

Elaine A. Liu, Mark L. Schultz, Chisaki Mochida, Chan Chung, Henry L. Paulson, and Andrew P. Lieberman. JCI Insight. 2020 Sep 15; doi.org/10.1172/jci.insight.136676.
 
MEF2 Impairment Underlies Skeletal Muscle Atrophy in Polyglutamine Disease
Samir R Nath, Matthew L Lieberman, Zhigang Yu, Caterina Marchioretti, Samuel T Jones, Emily C E Danby, Kate M Van Pelt, Gianni Sorarù, Diane M Robins, Gillian P Bates, Maria Pennuto, Andrew P Lieberman.
Acta Neuropathol. 2020 Apr 18;1-18. doi: 10.1007/s00401-020-02156-4.
 

Targeting Hsp70 facilitated protein quality control for treatment of polyglutamine diseases
Davis AK, Pratt WB, Lieberman AP, Osawa Y.  Cell Mol Life Sci. 2020 Mar;77(6):977-996.

2019

Schultz ML, Fawaz MV, Azaria RD, Hollon TC, Liu EA, Kunkel TJ, Halseth TA, Krus KL, Ming R, Morin EE, McLoughlin HS, Bushart DD, Paulson HL, Shakkottai VG, Orringer DA, Schwendeman AS, Lieberman AP.  BMC Med. 2019 Nov 11;17(1):200.
 
Ravi B, Antonellis A, Sumner CJ, Lieberman AP.  Hum Mol Genet. 2019 Oct 1;28(R1):R55-R64.
 
Eftekharzadeh B, Banduseela VC, Chiesa G, Martínez-Cristóbal P, Rauch JN, Nath SR, Schwarz DMC, Shao H, Marin-Argany M, Di Sanza C, Giorgetti E, Yu Z, Pierattelli R, Felli IC, Brun-Heath I, García J, Nebreda ÁR, Gestwicki JE, Lieberman AP, Salvatella X. Nat Commun. 2019 Aug 8;10(1):3562.
 
The intersection of lysosomal and endoplasmic reticulum calcium with autophagy defects in lysosomal diseases
Liu EA, Lieberman AP. Neurosci Lett. 2019 Apr 1; 697:10-16.

 

Polyglutamine Repeats in Neurodegenerative Diseases
Lieberman AP, Shakkottai VG, Albin RL. Annu Rev Pathol. 2019 Jan 24;14:1-27.

 

2018

Spinal and bulbar muscular atrophy
Lieberman AP. Handb Clin Neurol. 2018;148:625-632. doi: 10.1016/B978-0-444-64076-5.00040-5. Review.
 
Coordinate regulation of mutant NPC1 degradation by selective ER autophagy and MARCH6-dependent ERAD
Schultz ML, Krus KL, Kaushik S, Dang D, Chopra R, Qi L, Shakkottai VG, Cuervo AM, Lieberman AP. Nat Commun. 2018 Sep 10;9(1):3671. 
 
Nath SR, Yu Z, Gipson TA, Marsh GB, Yoshidome E, Robins DM, Todi SV, Housman DE, Lieberman AP. J Clin Invest. 2018 Aug 1;128(8):3630-3641.
 

2017

The Ubiquitination, Disaggregation and Proteasomal Degradation Machineries in Polyglutamine Disease
Nath SR, Lieberman AP. The Ubiquitination, Disaggregation and Proteasomal Degradation Machineries in Polyglutamine Disease. Front Mol Neurosci. 2017 Mar 22;10:78.doi: 10.3389/fnmol.2017.0007828003546.

 

2016

Giorgetti E, Yu Z, Chua JP, Shimamura R, Zhao L, Zhu F, Venneti S, Pennuto M, Guan Y, Hung G, Lieberman AP. Rescue of Metabolic Alterations in AR113Q Skeletal Muscle by Peripheral Androgen Receptor Gene Silencing. Cell Rep. 2016 Sep 27;17(1):125-36. doi: 10.1016/j.celrep.2016.08.084. Click here to read the paper. 

Giorgetti E, Lieberman AP. Polyglutamine androgen receptor-mediated neuromuscular disease. Cell Mol Life Sci. 2016 Nov;73(21):3991-9. Click here to read the paper. 

Schultz ML, Krus KL, Lieberman AP. Lysosome and endoplasmic reticulum quality control pathways in Niemann-pick type C disease. Brain Res. 2016 Mar 26. pii: S0006-8993(16)30166-4. Review. Click here  to read the paper.

Chung C1, Elrick MJ1, Dell'Orco JM2, Qin ZS3, Kalyana-Sundaram S1,4, Chinnaiyan AM1,4,5, Shakkottai VG2, Lieberman AP1. Heat Shock Protein Beta-1 Modifies Anterior to Posterior Purkinje Cell Vulnerability in a Mouse Model of Niemann-Pick Type C Disease. PLoS Genet. 2016 May 6;12(5):e1006042. doi: 10.1371/journal.pgen.1006042. eCollection 2016. Click here to read the paper.

Chung Chang, Puthanveetil P, Ory DS, Lieberman AP Genetic and pharmacological evidence implicates cathepsins in Niemann-Pick C cerebellar degeneration. Hum Mol Genet. 2016 Apr 1;25(7):1434-46. Epub 2016 Jan 28. Click here  to read the paper.

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1998-2015

Chua JP, Reddy SL, Yu Z, Giorgetti E, Montie HL, Mukherjee S, Higgins J, McEachin RC, Robins DM, Merry DE, Iñiguez-Lluhí JA, Lieberman AP. Disrupting SUMOylation enhances transcriptional function and ameliorates polyglutamine androgen receptor-mediated disease. The Journal of clinical investigation. 2015; 125(2):831-45. Click here  to read the paper.

Pratt WB, Gestwicki JE, Osawa Y, Lieberman AP.  Targeting Hsp90/Hsp70-based protein quality control for treatment of adult onset neurodegenerative diseases. Annual review of pharmacology and toxicology. 2015; 55:353-71. Click here  to read the paper.

Pratt WB, Morishima Y, Gestwicki JE, Lieberman AP, Osawa Y. A model in which heat shock protein 90 targets protein-folding clefts: rationale for a new approach to neuroprotective treatment of protein folding diseases. Experimental biology and medicine (Maywood, N.J.). 2014; 239(11):1405-13. Click here  to read the paper.

Lieberman AP, Yu Z, Murray S, Peralta R, Low A, Guo S, Yu XX, Cortes CJ, Bennett CF, Monia BP, La Spada AR, Hung G. Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy. Cell Rep. 2014 May 8;7(3):774-84.  Click here  to read the paper.

Chua JP, Reddy SL, Merry DE, Adachi H, Katsuno M, Sobue G, Robins DM, Lieberman AP. Transcriptional activation of TFEB/ZKSCAN3 target genes underlies enhanced autophagy in spinobulbar muscular atrophy. Hum Mol Genet, 23:1376-1386, 2014.  Click here to read the paper.

Chua JP, Lieberman AP. Pathogenic mechanisms and therapeutic strategies in spinobulbar muscular atrophy. CNS & Neurological Disorders – Drug Targets, 12:1146-1156, 2013.  Click here to read the paper.

Yu T, Lieberman AP. Npc1 acting in neurons and glia is essential for the formation and maintenance of CNS myelin. PLOS Genetics, 9:e1003462, 2013. Click here to read the paper.

Elrick MJ, Lieberman AP.  Autophagic dysfunction in a lysosomal storage disorder due to impaired proteolysis.  Autophagy.  2013 Feb 19:9(2) 234-235.  Click here to read the paper.

Wang AM, Miyata Y, Klinedinst S, Peng HM, Chua JP, Komiyama T, Li X, Morishima Y, Merry DE, Pratt, WB, Osawa Y, Collins CA, Gestwicki JE, Lieberman AP.  Activation of Hsp70 reduces neurotoxicity by promoting polyglutamine protein degradation.  Nat Chem Biol.  2013 (9) 112-118. doi: 10.1038/nchembio. 1140.  Click here to read the paper.

Elrick MJ, Yu T, Chung C, Lieberman AP.  Impaired proteolysis underlies autophagic dysfunction in Niemann-Pick type C disease.  Hum Mol Genet. 2012 Nov 15;21(22):4876-87. doi: 10.1093/hmg/dds324. Epub 2012 Aug 7.  Click here to read the paper.

Yu T, Chung C, Shen D, Xu H, Lieberman AP.  Ryanodine receptor antagonists adapt NPC1 proteostasis to ameliorate lipid storage in Niemann-Pick type C disease fibroblasts.  Hum Mol Genet. 2012 Jul 15;21(14):3205-14. doi: 10.1093/hmg/dds145. Epub 2012 Apr 14.  Click here to read the paper.

Lieberman AP, Puertollano R, Raben N, Slaugenhaupt S, Walkley SU, Ballabio A.  Autophagy in lysosomal storage disorders.  Autophagy. 2012 May 1;8(5):719-30. doi: 10.4161/auto.19469. Epub 2012 May 1. Review.  Click here to read the paper.

Yu Z, Wang A M, Adachi H, Katsuno M, Sobue G, Yue Z, Robins D M, Lieberman A P.  Macroautophagy is regulated by the UPR-mediator CHOP and accentuates the phenotype of SBMA mice.  PLoS Genet. 2011 Oct; 7(10):e1002321. Click here to read the paper.

Yu T, Shakkottai V G, Chung C, Lieberman A P.  Temporal and cell-specific deletion establishes that neuronal Npc1 deficiency is sufficient to mediate neurodegeneration.  Hum Mol Genet.  2011 Nov 15;20(22):4440-51. Click here to read the paper.

Elrick M J, Pacheco C D, Yu T, Dadgar N, Shakkottai V G, Ware C, Paulson H L, Lieberman A P. Conditional Niemann-Pick C mice demonstrate cell autonomous Purkinje cell neurodegeneration.  Hum Mol Genet, 19:837-847, 2010. Click here to read the paper.

Wang A M, Morishima Y, Clapp K M, Peng H M, Pratt W B, Gestwicki J E, Lieberman A P. Inhibition of Hsp70 by methylene blue affects signaling protein function and ubiquitination and modulates polyglutamine protein degradation.  J Biol Chem, 285:15714-15723, 2010. Click here to read the paper.

Yu Z, Wang A M, Robins D M, Lieberman A P. Altered RNA splicing contributes to skeletal muscle pathology in Kennedy disease knock-in mice. Dis Model Mech. 2009 Sep-Oct;2(9-10):500-7. Click here to read the paper.

Pacheco C D, Elrick M J, Lieberman A P. Tau normal function influences Niemann-Pick type C disease pathogenesis in mice and modulates autophagy in NPC1-deficient cells.  Autophagy. 2009 May;5(4):548-50. Click here to read the paper.

Pacheco C D, Elrick M J, Lieberman A P. Tau deletion exacerbates the phenotype of Niemann-Pick type C mice and implicates autophagy in pathogenesis.  Hum Mol Genet. 2009 Mar 1;18(5):956-65. Click here to read the paper.

Jordan C L, Lieberman A P. Spinal and bulbar muscular atrophy: A motorneuron or muscle disease? Current Opinion Pharmacology. Click here to read the paper.

Pacheco C D, Lieberman A P. The pathogenesis of Niemann-Pick type C disease: A role for autophagy? Expert Rev Mol Med. Click here to read the paper.

Morishima Y, Wang A M, Yu Z, Pratt W B, Osawa Y, Lieberman A P. CHIP deletion reveals functional redundancy of E3 ligases in promoting degradation of both signaling proteins and expanded glutamine proteins. Hum Mol Genet. Click here to read the paper.

Lieberman A P, Robins D M. The androgen receptor’s CAG/glutamine tract in mouse models of neurological disease and cancer. J Alzheimer’s Dis,14:247-255, 2008. Click here to read the paper.

Pacheco C D, Kunkel R, Lieberman A P. Autophagy in Niemann-Pick C disease is Beclin-1 dependent and responsive to lipid trafficking defects. Hum Mol Genet, 16, 1495-1503, 2007. Click here to read the paper.

Yu Z, Dadgar N, Albertelli M, Gruis K, Jordan C, Robins D M, Lieberman A P. Androgen-dependent pathology demonstrates myopathic contribution to the kennedy disease phenotype in a mouse knock-in model. J Clin Invest, 116,2663-2673, 2007. Click here to read the paper.

Thomas M, Harrell J M, Morishima Y, Peng H M, Pratt W B, Lieberman A P. Pharmacologic and genetic inhibition of hsp90-dependent trafficking reduces aggregation and promotes degradation of the expanded glutamine androgen receptor without stress protein induction. Hum Mol Genet, 15:1876-83, 2006. Click here to read the paper.

Yu Z, Dadgar N, Albertelli M, Scheller A, Albin R L, Robins D M, Lieberman A P. Abnormalities of germ cell maturation and sertoli cell cytoskeleton in androgen receptor 113 CAG knock-in mice reveal toxic effects of the mutant protein. Am J Pathol, 168:195-204, 2006. Click here to read the paper.

Thomas M, Yu Z, Dadgar N, Varambally S, Yu J, Chinnaiyan A M, Lieberman A P. The unfolded protein response modulates toxicity of the expanded glutamine androgen receptor. J Biol Chem, 280:21264-21271, 2005. Click here to read the paper.

Thomas M, Dadgar N, Aphale A, Harrell J M, Kunkel R, Pratt W B, Lieberman A P. Androgen receptor acetylation site mutations cause trafficking defects, misfolding and aggregation similar to expanded glutamine tracts. J Biol Chem, 279:8389-8395, 2004. Click here to read the paper.

Lieberman A P. SUMO, a ubiquitin-like modifier implicated in neurodegeneration. Exp Neurol, 185:204-207, 2004. Click here to read the paper.

Lieberman A P, Harmison G, Strand A D, Olson J M, Fischbeck K H. Altered transcriptional regulation in cells expressing the expanded polyglutamine androgen receptor. Hum Mol Genet, 11:1967-1976, 2002. Click here to read the paper.

Lieberman A P, Puls I, Fischbeck K H. Mining the genome for causes and cures of neurological disease. Trends Pharmacol Sci, 22:161-162, 2001. Click here to read the paper.

Lieberman A P, Fischbeck K H. Triplet repeat expansion in neuromuscular disease. Muscle & Nerve, 23:843-850, 2000. Click here to read the paper.

Lieberman A P, Trojanowski J Q, Leonard D G B, Chen K-L, Barnett J L, Leverenz J B, Bird T D, Robitaille Y, Malandrini A, Fischbeck K H. Ataxin 1 and ataxin 3 in neuronal intranuclear inclusion disease. Ann Neurol, 46:271-273, 1999. Click here to read the paper.

Lieberman A P, Robitaille Y, Trojanowski J Q T, Dickson D W, and Fischbeck K H. Polyglutamine-containing aggregates in neuronal intranuclear inclusion disease. Lancet, 351:884, 1998. Click here to read the paper.