Published April 25, 2025 | Version v1
Dataset Open

Accelerating Drug Repurposing for Rett Syndrome Project

Creators

  • 1. ROR icon University of Alabama at Birmingham

Contributors

Data curator:

Data manager:

  • 1. ROR icon University of Alabama at Birmingham

Description

Above are the datasets that were pulled from PharmAlchemy, BindingDB, and other databases, and then inspected and curated with the assistance of AI Agents to identify potential candidate drugs for repurposing regarding Rett Syndrome. 

Also included is the project presentation, Final Report, and data from Perplexity and AIScholar.

SQL Coding performed in PharmAlchemy Oracle APEX is also shown

Files

AI2SCHOLAR.pdf

Files (4.6 MB)

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Additional details

Identifiers

Other
N/A

Related works

Is supplemented by
Dataset: PharmAlchemy (Other)

Dates

Created
2025-05-01
Created in Zenodo

Software

Repository URL
https://github.com/enyarat/rett-drug-repurposing.git
Programming language
SQL
Development Status
Active

References

  • Bello, O., Blair, K., Chapleau, C., & Larimore, J. L. (2013). Is memantine a potential therapeutic for Rett syndrome?Frontiers in Neuroscience, 7, 245. https://doi.org/10.3389/fnins.2013.00245
  • Blank, C. (2023, March 13). FDA approves first treatment for Rett syndrome. Managed Healthcare Executive. https://www.managedhealthcareexecutive.com/view/fda-approves-first-treatment-for-rett-syndrome
  • Buntz, B. (2024, December 3). Why scientific AI needs clear lines of sight — especially for fields like drug development. Drug Discovery & Development. https://www.drugdiscoverytrends.com/why-scientific-ai-needs-clear-lines-of-sight-especially-for-fields-like-drug-development/
  • Castro, J., Garcia, R. I., Kwok, S., Banerjee, A., Petravicz, J., Woodson, J., Mellios, N., … Sur, M. (2014). Functional recovery with recombinant human IGF1 treatment in a mouse model of Rett syndrome. Proceedings of the National Academy of Sciences, 111(27), 9941–9946. https://doi.org/10.1073/pnas.1311685111
  • Christodoulou, J., Krishnaraj, R., & Orlic-Milacic, M. (2025). Transcriptional Regulation by MECP2 (R-HSA-8986944). Reactome, European Bioinformatics Institute, New York University Langone Medical Center, Ontario Institute for Cancer Research, Oregon Health and Science University. https://reactome.org/content/detail/R-HSA-8986944
  • Fogarty, M. J. (2023). Inhibitory synaptic influences on developmental motor disorders. International Journal of Molecular Sciences, 24(8), 6962. https://doi.org/10.3390/ijms24086962:contentReference[oaicite:1]{index=1}
  • Gonzales, M. L., & LaSalle, J. M. (2010). The role of MeCP2 in brain development and neurodevelopmental disorders. Current Psychiatry Reports, 12(2), 127–134. https://doi.org/10.1007/s11920-010-0097-7
  • Knox, C., Wilson, M., Klinger, C. M., Franklin, M., Oler, E., Wilson, A., … Wishart, D. S. (2024). DrugBank 6.0: The DrugBank knowledgebase for 2024. Nucleic Acids Research, 52(D1), D1265–D1275. https://doi.org/10.1093/nar/gkad976
  • Liu, T., Hwang, L., Burley, S. K., Nitsche, C. I., Southan, C., Walters, W. P., & Gilson, M. K. (2024). BindingDB in 2024: A FAIR knowledgebase of protein–small-molecule binding data. Nucleic Acids Research, 53(D1), D1633–D1641. https://doi.org/10.1093/nar/gkae1075
  • Milacic, M., Beavers, D., Conley, P., Gong, C., Gillespie, M., Griss, J., Haw, R., Jassal, B., Matthews, L., May, B., Petryszak, R., Ragueneau, E., Rothfels, K., Sevilla, C., Shamovsky, V., Stephan, R., Tiwari, K., Varusai, T., Weiser, J., … D'Eustachio, P. (2024). The Reactome Pathway Knowledgebase 2024. Nucleic Acids Research, 52(D1), D672–D678. https://doi.org/10.1093/nar/gkad1025
  • Pini, G., Congiu, L., Benincasa, A., DiMarco, P., Bigoni, S., Dyer, A. H., Mortimer, N., Della-Chiesa, A., O'Leary, S., McNamara, R., Mitchell, K. J., Gill, M., & Tropea, D. (2016). Illness severity, social and cognitive ability, and EEG analysis of ten patients with Rett syndrome treated with Mecasermin (recombinant human IGF-1). Autism Research and Treatment, 2016, Article 5073078. https://doi.org/10.1155/2016/5073078
  • Piñero, J., Ramírez-Anguita, J. M., Sauch-Pitarch, J., Ronzano, F., Centeno, E., Sanz, F., & Furlong, L. I. (2020). DisGeNET: A comprehensive platform integrating information on human disease-associated genes and variants. Nucleic Acids Research, 48(D1), D845–D855. https://doi.org/10.1093/nar/gkz1021
  • PharmAlchemy Team. (2024). PharmAlchemy: Uncovering Disease - Drug - Gene Relationships. PharmAlchemy2024_FinalPaper. University of Alabama at Birmingham.
  • The UniProt Consortium. (2023). UniProt: The universal protein knowledgebase in 2023. Nucleic Acids Research, 51(D1), D523–D531. https://doi.org/10.1093/nar/gkac1052
  • Wang, H., Pati, S., Pozzo-Miller, L., & Doering, L. C. (2015). Targeted pharmacological treatment of autism spectrum disorders: Fragile X and Rett syndromes. Frontiers in Cellular Neuroscience, 9, Article 55. https://doi.org/10.3389/fncel.2015.00055
  • Wang, H., Westmark, C. J., Frost, E., & Doering, L. C. (2012). Recent progress in understanding plasticity in neurogenetic disorders. Neural Plasticity, 2012, 812472. https://doi.org/10.1155/2012/812472
  • Zdrazil, B., Felix, E., Hunter, F., Manners, E. J., Blackshaw, J., Corbett, S., … Leach, A. R. (2024). The ChEMBL database in 2023: A drug discovery platform spanning multiple bioactivity data types and time periods. Nucleic Acids Research, 52(D1), D1180–D1192. https://doi.org/10.1093/nar/gkad1004