Zinc-Finger Nucleases: the past and future of in vivo genome editing

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The race to discover the solution to genetic disease has been a long one; only recently has it come to look like it is a race that may actually be won. Zinc-Finger Nucleases (ZFNs) are synthetic enzymes that can be designed to generate targeted double-stranded breaks in DNA, giving the scientific community a way to effectively stimulate genomic changes. With the exquisite control granted by the use of custom ZFN technology, the door has been opened to the age of in vivo genome editing. Through examination of the development of this synthetic nuclease technology, as well as its past and present uses, conjecture can be made regarding future use. This study uses Cystic Fibrosis as a model to which ZFNs may be applied, examining the possibility of using this technology as a treatment for this disease in the future.
Thesis completed in partial fulfillment of the requirements for the Alfred University Honors Program.
Honors thesis, Genetics, Zinc-Finger Nucleases, Enzymes, Cystic fibrosis