Rachel Bailey, Ph.D. Titles and Appointments Assistant Professor Schools Medical School | Graduate School Departments Center for Alzheimer's and Neurodegenerative Diseases | Pediatrics Graduate Programs Neuroscience Biography Dr. Bailey received dual Bachelor of Science degrees in Biology/Bioinformatics and Molecular Biology from Rensselaer Polytechnic Institute. She began her Ph.D. in Neuroscience at the Mayo Clinic and completed it at the University of Florida under the mentorship of Dr. Jada Lewis. Dr. Bailey’s graduate work focused on studying modifiers of tauopathies. She demonstrated that the LRRK2 protein that is linked to Parkinson’s disease directly phosphorylates tau protein at a novel epitope, which is an important step in the neurodegenerative process. For her postdoctoral fellowship, Dr. Bailey worked in the Gene Therapy Center at the University of North Carolina Chapel Hill in the lab of Dr. Steven Gray. She contributed to the preclinical development of an AAV-based gene therapy for the rare pediatric neurodegenerative disorder, Giant Axonal Neuropathy (GAN), which is currently being tested in a Phase I clinical trial that is ongoing at the Clinical Center at the NIH (NCT02362438). Dr. Bailey also characterized autonomic nervous system dysfunction in GAN rodent models and optimized targeting AAV vector to the peripheral nervous system in these models. Now at UT Southwestern, the research in the Bailey lab focuses on the development of gene therapies for neurological disorders and facilitates the translation of these treatments for use in human. Pediatric disorders that she is working on include SLC13A5 epileptic encephalopathy, Multiple Sulfatase Deficiency, Charcot Marie Tooth disease type 4J and GAN. Dr. Bailey has expanded her gene therapy research efforts beyond monogenic diseases to more complex neurodegenerative disorders, including tauopathies, such as Alzheimer’s disease. Her lab utilizes AAV vector engineering for both gene replacement and gene-silencing approaches, explores alternative methods of virus delivery, performs proof-of-concept studies in cell culture models, and performs IND-enabling studies in animal models for translation of treatments to the clinic. Research Interest Developing gene therapies for Alzheimer's and neurodegenerative diseases Developing gene therapies for pediatric neurological disorders Engineering adeno-associated viral (AAV) vectors Selective targeting of AAV vectors to the central and peripheral nervous systems Translation of gene therapies to clinical trials 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 Communications Medicine 2025 Dec 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 Muscle and Nerve 2025 Nov 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 Gene Therapy 2025 Jul 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 Developmental Medicine and Child Neurology 2025 Jul 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 Gene Therapy 2025 May 32 189-196 Direct Vagus Nerve Injection Protocol For Rats Regmi NL, Chen X, Bailey RM, Gray SJ Journal of Visualized Experiments 2025 Mar 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 Brain Communications 2025 7 Toward a translational gene therapy for mucolipidosis IV Snyder B, Bailey RM Molecular Therapy Methods and Clinical Development 2024 Dec 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 Genes 2024 Oct 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 New England Journal of Medicine 2024 Mar 390 1092-1104 Results 1-10 of 30 1 2 3 Next Last Books Featured Books Methods for Gene Transfer to the Central Nervous System. In Advances in Genetics Kantor B, Bailey RM, Wimberly K, Nagabhushan Kalburgi S, and Gray SJ. (2014). Cambridge, Massachusetts, Academic Press Honors & Awards Finalist in Swim with the Sharks!A Team Science Pilot Grant Competition, UT Southwestern (2018) NIH NRSA Postdoctoral Fellowship (2016) American Society for Gene and Cell TherapyConference Travel Scholarship (2015) NIH T32 Postdoctoral Fellowship (2015) Peripheral Nerve Society AwardTravel Scholarship (2015) Bryan Robinson Endowment (2013) NIH NRSA Pre-doctoral Fellowship (2012) Parkinson's Disease Summer Student Fellowship (2012) Robert D. and Patricia E. Kern Pre-doctoral Fellowship (2008) Professional Associations/Affiliations American Society for Gene and Cell Therapy (2014) International Society to Advance Alzheimer's Research and Treatment Peripheral Nerve Society Society for Neuroscience (2012)
