Date of Award

6-8-2015

Document Type

Thesis

Publisher

Santa Clara University

First Advisor

Leilani Miller

Abstract

Genetic engineering is an emerging technology that offers the potential to prevent, treat, or cure genetic diseases. The technology can permanently alter the genome, providing an alternative therapy to drugs and surgery. Specifically, gene therapy is a promising treatment option for many incurable genetic diseases, such as cystic fibrosis and muscular cell dystrophy. Our project gives rise to a better understanding of TALENs and its uses in the genetic engineering field. TALENs, transcription activator-like effector nucleases, are a genetic engineering technology that can be used for targeted gene modification. They are engineered proteins that can bind to specific sequences of DNA and induce a double-stranded break. The DNA sequence that the TALENs bind to is determined by the user; therefore the TALENs can be engineered to target specific DNA sequences that cause genetic diseases. We used TALENs within the model organism, nematode C. elegans, to explore their potential for use in gene therapy. By utilizing TALENs to introduce a lin-31 mutant into the genome of C. elegans we aim to advance the understanding of TALENs as a genetic engineering tool and contribute to the research on the docking site of LIN-31 in the Ras/MAPK signaling pathway. Our group was successful in creating the DNA that encodes for these TALENs proteins, providing a foundation for future student researchers to continue on the project.

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