Honors College Thesis

 

Nucleic Acid Based Therapeutics for Treatment of Cystic Fibrosis Public Deposited

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https://ir.library.oregonstate.edu/concern/honors_college_theses/z316q817j

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  • Cystic fibrosis is an inherited genetic disease caused by a single gene mutation in the CFTR protein. Affecting 70,000 people worldwide, this disease is debilitating to patients, resulting in persistent lung infections and decreased quality of life. Due to the various mutation types, there is not currently a single drug available to treat all forms of this disease. This has made this disease attractive for gene therapy. Currently, studies are being performed using viral, and nonviral treatment methods. This paper reviews current gene therapy treatments for this disease, and details specific results regarding lipid nanoparticle delivery of CFTR mRNA for treatment of Cystic Fibrosis.
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