Publications

Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency

Presa M, Bailey RM, Ray S, Bailey L, Tata S, Murphy T, Piec PA, Combs H, Gray SJ, Lutz C 2025 Dec Communications Medicine 5

T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)

Armao D, Bouldin TW, Bharucha-Goebel DX, Hartman TS, Gray SJ, Bailey RM, Saade D, Todd JJ, Jain M, Waite M, Bönnemann CG, Smith JK 2025 Nov Muscle and Nerve 72 1152-1155

Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus

Bhardwaj D, Youssef I, Imphean D, Holmes SK, Krishnan V, Estill-Terpack SJ, Diamond M, Chopra R, Bailey RM, Shah BR 2025 Jul Gene Therapy 32 376-384

Developmental phenotype and quality of life in SLC13A5 citrate transporter disorder

Ozlu C, Adams RM, Solidum RM, Cooper S, Best CR, Elacio J, Kavanaugh BC, Spelbrink EM, Brown TL, Nye K, Liu JS, Bailey RM, Goodspeed K, Porter BE 2025 Jul Developmental Medicine and Child Neurology 67 930-940

Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice

Garza IT, Eller MM, Holmes SK, Schackmuth MK, Bailey RM 2025 May Gene Therapy 32 189-196

Direct Vagus Nerve Injection Protocol For Rats

Regmi NL, Chen X, Bailey RM, Gray SJ 2025 Mar Journal of Visualized Experiments 2025-March

Neurological, metabolic and inflammatory phenotypes in a mouse model of ECHS1 deficiency

Eller MM, Zuberi AR, Fu X, Montalbano AP, Nitschke F, Burgess SC, Lutz C, Bailey RM 2025 Brain Communications 7

Toward a translational gene therapy for mucolipidosis IV

Snyder B, Bailey RM 2024 Dec Molecular Therapy Methods and Clinical Development 32

Sleep Abnormalities in SLC13A5 Citrate Transporter Disorder

Adams RM, Ozlu C, Bailey LE, Solidum RM, Cooper S, Best CR, Elacio J, Kavanaugh BC, Brown TL, Nye K, Liu J, Porter BE, Goodspeed K, Bailey RM 2024 Oct Genes 15

Intrathecal Gene Therapy for Giant Axonal Neuropathy

Bharucha-Goebel DX, Todd JJ, Saade D, Norato G, Jain M, Lehky T, Bailey RM, Chichester JA, Calcedo R, Armao D, Foley AR, Mohassel P, Tesfaye E, Carlin BP, Seremula B, Waite M, Zein WM, Huryn LA, Crawford TO, Sumner CJ, Hoke A, Heiss JD, Charnas L, Hooper JE, Bouldin TW, Kang EM, Rybin D, Gray SJ, Bönnemann CG 2024 Mar New England Journal of Medicine 390 1092-1104

SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy

Goodspeed K, Liu JS, Nye KL, Prasad S, Sadhu C, Tavakkoli F, Bilder DA, Minassian BA, Bailey RM 2022 Sep Genes 13

Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

Goodspeed K, Bailey RM, Prasad S, Sadhu C, Cardenas JA, Holmay M, Bilder DA, Minassian BA 2022 Jun Frontiers in Neurology 13

Gene Transfer Therapy for Neurodevelopmental Disorders

Ozlu C, Bailey RM, Sinnett S, Goodspeed KD 2021 Sep Developmental Neuroscience 43 230-240

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

Presa M, Bailey RM, Davis C, Murphy T, Cook J, Walls R, Wilpan H, Bogdanik L, Lenk GM, Burgess RW, Gray SJ, Lutz C 2021 Jun Journal of Clinical Investigation 131

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease

Armao D, Bouldin TW, Bailey RM, Gray SJ 2021 Ophthalmic Genetics 42 600-603

Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

Bailey RM, Rozenberg A, Gray SJ 2020 Jul Brain Research 1739

Advancing the pathologic phenotype of giant axonal neuropathy: Early involvement of the ocular lens

Armao D, Bouldin TW, Bailey RM, Hooper JE, Bharucha DX, Gray SJ 2019 Feb Orphanet Journal of Rare Diseases 14

Lrrk promotes tau neurotoxicity through dysregulation of actin and mitochondrial dynamics

Bardai FH, Ordonez DG, Bailey RM, Hamm M, Lewis J, Feany MB 2018 Dec PLoS biology 16

Physiologically relevant factors influence tau phosphorylation by leucine-rich repeat kinase 2

Hamm M, Bailey R, Shaw G, Yen SH, Lewis J, Giasson BI 2015 Oct Journal of Neuroscience Research 93 1567-1580

Effects of the C57BL/6 strain background on tauopathy progression in the rTg4510 mouse model

Bailey RM, Howard J, Knight J, Sahara N, Dickson DW, Lewis J 2014 Jan Molecular neurodegeneration 9

LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

Bailey RM, Covy JP, Melrose HL, Rousseau L, Watkinson R, Knight J, Miles S, Farrer MJ, Dickson DW, Giasson BI, Lewis J 2013 Dec Acta Neuropathologica 126 809-827

In vivo functional brain mapping in a conditional mouse model of human tauopathy (tau p301l) reveals reduced neural activity in memory formation structures

Perez PD, Hall G, Kimura T, Ren Y, Bailey RM, Lewis J, Febo M, Sahara N 2013 Molecular neurodegeneration 8

Neuronal sensitivity to TDP-43 overexpression is dependent on timing of induction

Cannon A, Yang B, Knight J, Farnham IM, Zhang Y, Wuertzer CA, D'Alton S, Lin WL, Castanedes-Casey M, Rousseau L, Scott B, Jurasic M, Howard J, Yu X, Bailey R, Sarkisian MR, Dickson DW, Petrucelli L, Lewis J 2012 Jun Acta Neuropathologica 123 807-823

CHIP regulates leucine-rich repeat kinase-2 ubiquitination, degradation, and toxicity

Ko HS, Bailey R, Smith WW, Liu Z, Shin JH, Lee YI, Zhang YJ, Jiang H, Ross CA, Moore DJ, Patterson C, Petrucelli L, Dawson TM, Dawson VL 2009 Feb Proceedings of the National Academy of Sciences of the United States of America 106 2897-2902

Akt and CHIP coregulate tau degradation through coordinated interactions

Dickey CA, Koren J, Zhang YJ, Xu YF, Jinwal UK, Birnbaum MJ, Monks B, Sun M, Cheng JQ, Patterson C, Bailey RM, Dunmore J, Soresh S, Leon C, Morgan D, Petrucelli L 2008 Mar Proceedings of the National Academy of Sciences of the United States of America 105 3622-3627

Organic anion transporter 3 (Oat3/Slc22a8) knockout mice exhibit altered clearance and distribution of penicillin G

VanWert AL, Bailey RM, Sweet DH 2007 Oct American Journal of Physiology - Renal Physiology 293 F1332-F1341

Identification of potential protein interactors of Lrrk2

Dächsel JC, Taylor JP, Mok SS, Ross OA, Hinkle KM, Bailey RM, Hines JH, Szutu J, Madden B, Petrucelli L, Farrer MJ 2007 Oct Parkinsonism and Related Disorders 13 382-385

Progranulin mediates caspase-dependent cleavage of TAR DNA binding protein-43

Zhang YJ, Xu YF, Dickey CA, Buratti E, Baralle F, Bailey R, Pickering-Brown S, Dickson D, Petrucelli L 2007 Sep Journal of Neuroscience 27 10530-10534

The high-affinity HSP90-CHIP complex recognizes and selectively degrades phosphorylated tau client proteins

Dickey CA, Kamal A, Lundgren K, Klosak N, Bailey RM, Dunmore J, Ash P, Shoraka S, Zlatkovic J, Eckman CB, Patterson C, Dickson DW, Nahman NS, Hutton M, Burrows F, Petrucelli L 2007 Mar Journal of Clinical Investigation 117 648-658

The prolyl hydroxylase oxygen-sensing pathway is cytoprotective and allows maintenance of mitochondrial membrane potential during metabolic inhibition

Sridharan V, Guichard J, Bailey RM, Kasiganesan H, Beeson C, Wright GL 2007 Feb American Journal of Physiology - Cell Physiology 292 C719-C728