Skip to Main Content
 

Global Search Box

 
 
 
 

Files

File List


Final Thesis.pdf (6.64 MB)

Digital Accessibility Report

File List


Final Thesis.pdf.accreport.html (7.81 KB)

ETD Abstract Container

Abstract Header

Repurposing of Zoledronic Acid for Treatment of Duchenne Muscular Dystrophy

Haroon, Alexa Grey
http://rave.ohiolink.edu/etdc/view?acc_num=ouhonors1714146914719856

Abstract Details

2024, Bachelor of Science (BS), Ohio University, Neuroscience.
Duchene muscular dystrophy (DMD) is an X-Linked recessive genetic disorder which occurs in approximately 1/5000 XY births and is caused by a mutation in the human dystrophin gene. DMD causes many physiological defects and drastically shortens the lifespan of those afflicted by it. Gene replacement therapies are in clinical trials, but traditional therapies are still needed whilst those are developed. The C. elegans dys-1 gene is highly conserved compared to human dystrophin, and mutations in C. elegans dys-1 produces a clinically relevant phenotype. By use of the C. elegans DMD model, the pathology of DMD can easily be studied in a laboratory setting, allowing various potential traditional treatments to be tested for effectiveness. Zoledronic Acid (ZA) is approved by the U.S. Food and Drug Administration (FDA) to treat osteoporosis in patients with DMD, and preliminary data suggests that ZA could have positive effects for muscular health in DMD patients without osteoporosis. In the present work, it was found that the drug ZA is effective in improving DMD health in the C. elegans DMD model. Additionally, it was found that ZA improves DMD health through lowering the increased Ca2+ levels classically found in dys-1 mutants, which along with further experimentation informs a potential mechanistic pathway through which ZA acts. With this data, it is hoped that a clinical trial for repurposing ZA for use in for DMD patients is conducted in order to lessen the suffering caused by this disorder whilst gene therapeutics are developed.
Nathaniel Szewczyk (Advisor)
Corinne Nielsen (Advisor)
50 p.
Neurosciences
Duchenne muscular dystrophy; DMD; C. elegans; Zoledronic Acid; ZA; Neuroscience; Degeneration; Drug repurposing

Recommended Citations

Citations

  • Haroon, A. G. (2024). Repurposing of Zoledronic Acid for Treatment of Duchenne Muscular Dystrophy [Undergraduate thesis, Ohio University]. OhioLINK Electronic Theses and Dissertations Center. http://rave.ohiolink.edu/etdc/view?acc_num=ouhonors1714146914719856

    APA Style (7th edition)

  • Haroon, Alexa. Repurposing of Zoledronic Acid for Treatment of Duchenne Muscular Dystrophy. 2024. Ohio University, Undergraduate thesis. OhioLINK Electronic Theses and Dissertations Center, http://rave.ohiolink.edu/etdc/view?acc_num=ouhonors1714146914719856.

    MLA Style (8th edition)

  • Haroon, Alexa. "Repurposing of Zoledronic Acid for Treatment of Duchenne Muscular Dystrophy." Undergraduate thesis, Ohio University, 2024. http://rave.ohiolink.edu/etdc/view?acc_num=ouhonors1714146914719856

    Chicago Manual of Style (17th edition)

ouhonors1714146914719856
124
© 2024, all rights reserved.
This open access ETD is published by Ohio University Honors Tutorial College and OhioLINK.