Faculty & Staff

Barbara Bailus, PhD

The Bailus Lab focuses on neurological disorders, with a specific emphasis on gene therapy and delivery mechanisms. We build custom proteins in the lab that have the ability to cross the blood-brain barrier and have a therapeutic effect. These therapeutic proteins include gene editing enzymes and other various proteins. Some of the diseases and disorders studied in the lab include Angelman syndrome, SETBP1 Haploinsufficiency Disorder, Huntington’s disease, and Alzheimer’s disease. During her doctoral studies, Dr. Bailus was the first to publish on a Zinc Finger construct crossing the blood brain barrier in mice, due to the attachment of a cell penetrating peptide. During her postdoctoral studies, Dr. Bailus was the first to publish on the link between the protein FKBP51 and Huntington’s disease. The Bailus Lab continues to work on discovering and characterizing cell-penetrating peptides for the delivery of therapeutics. Dr. Bailus has presented her work at many National and International conferences, including ASGCT. Dr. Bailus has been honored to be the recipient of a New Investigator grant from the Foundation for Angelman Syndrome Therapeutics.

  1. Bailus BJ, Segal DJ. The prospect of molecular therapy for Angelman syndrome and other monogenic neurologic disorders. BMC Neurosci. 2014 Jun 19;15:76. doi: 10.1186/1471-2202-15-76. PMID: 24946931; PMCID: PMC4069279.
  2. Bailus BJ, Scheeler SM, Simons J, Sanchez MA, Tshilenge KT, Creus-Muncunill J, Naphade S, Lopez-Ramirez A, Zhang N, Lakshika Madushani K, Moroz S, Loureiro A, Schreiber KH, Hausch F, Kennedy BK, Ehrlich ME, Ellerby LM. Modulating FKBP5/FKBP51 and autophagy lowers HTT (huntingtin) levels. Autophagy. 2021 Dec;17(12):4119-4140. doi: 10.1080/15548627.2021.1904489. Epub 2021 May 24. PMID: 34024231; PMCID: PMC8726715.
  3. Ring KL, An MC, Zhang N, O'Brien RN, Ramos EM, Gao F, Atwood R, Bailus BJ, Melov S, Mooney SD, Coppola G, Ellerby LM. Genomic Analysis Reveals Disruption of Striatal Neuronal Development and Therapeutic Targets in Human Huntington's Disease Neural Stem Cells. Stem Cell Reports. 2015 Dec 8;5(6):1023-1038. doi: 10.1016/j.stemcr.2015.11.005. PMID: 26651603; PMCID: PMC4682390.
  4. Bailus B, Zhang N, Ellerby LM. Using Genome Engineering to Understand Huntington’s Disease. 2017 Sep 15. In: Jaenisch R, Zhang F, Gage F, editors. Genome Editing in Neurosciences [Internet]. Cham (CH): Springer; 2017. PMID: 31314441.
  5. Zhang N, Bailus BJ, Ring KL, Ellerby LM. iPSC-based drug screening for Huntington's disease. Brain Res. 2016 May 1;1638(Pt A):42-56. doi: 10.1016/j.brainres.2015.09.020. Epub 2015 Sep 30. PMID: 26428226; PMCID: PMC4814369.

Barbara Bailus’ complete research publications can be found at: https://pubmed.ncbi.nlm.nih.gov/?term=Barbara+Bailus.

The Bailus Lab focuses on neurological disorders, with a specific emphasis on gene therapy and delivery mechanisms. We build custom proteins in the lab that have the ability to cross the blood-brain barrier and have a therapeutic effect. These therapeutic proteins include gene editing enzymes and other various proteins. Some of the diseases and disorders studied in the lab include Angelman syndrome, SETBP1 Haploinsufficiency Disorder, Huntington’s disease, and Alzheimer’s disease. During her doctoral studies, Dr. Bailus was the first to publish on a Zinc Finger construct crossing the blood brain barrier in mice, due to the attachment of a cell penetrating peptide. During her postdoctoral studies, Dr. Bailus was the first to publish on the link between the protein FKBP51 and Huntington’s disease. The Bailus Lab continues to work on discovering and characterizing cell-penetrating peptides for the delivery of therapeutics. Dr. Bailus has presented her work at many National and International conferences, including ASGCT. Dr. Bailus has been honored to be the recipient of a New Investigator grant from the Foundation for Angelman Syndrome Therapeutics.