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How CRISPR/Cas9 Can Be Used to Treat Cystic Fibrosis

Project by Polygence alum Nandita

How CRISPR/Cas9 Can Be Used to Treat Cystic Fibrosis

Project's result

Created a Review Paper discussing CRISPR/Cas9, how CRISPR/Cas9 can be used in the treatment of Cystic Fibrosis, and the ethics of such treatments.

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Summary

From its discovery in the immune systems of prokaryotes, the clustered regularly interspaced short palindromic repeats/CRISPR-associated (CRISPR/Cas9) system has revolutionized gene editing. With these advances in gene editing, there come many potential medical applications of the CRISPR/Cas9 technology to treat genetic diseases. This paper describes methods of using CRISPR/Cas9 to treat the genetic disorder Cystic Fibrosis (CF). CF is caused by a mutated cystic fibrosis transmembrane conductance regulator (CFTR) gene, affecting many of the body’s cells. People carrying this mutation have thick and sticky mucus which can build up in the digestive tract and lungs, leading to severe blockages and damage. These blockages can lead to trouble breathing and a higher possibility of infection. This paper examines the current cystic fibrosis treatment, including the available drugs and their limitations. CRISPR/Cas9 has the potential to completely cure the disease in one treatment through the modification of the CFTR gene in the patients’ cells. This paper describes delivery mechanisms and mechanistic details of the gene editing process and how it can be applied to cystic fibrosis patients.

Jacob

Jacob

Polygence mentor

PhD Doctor of Philosophy candidate

Subjects

Medicine, Biology

Expertise

Biology, Biochemistry, Genetics, Bioethics

Nandita

Nandita

Student

School

Oak Park High School

Graduation Year

2024

Project review

“I really loved being able to work with a mentor! What really helped me was that my mentor would teach me about the topic. I came in with a vague idea of the topic, and I assumed I'd be on my own for all my research. What really happened was that my mentor would ask me to look at a paper myself, and then we'd talk about it in our next session. That was really amazing for me, because I was able to test what I knew and try to understand everything on my own first. Then, my mentor gave me an in depth explanation on anything I was confused about. I came out of each session with a much deeper understanding of gene therapy and Cystic Fibrosis. What I really wanted out of Polygence was to learn, and I definitely got that!”

About my mentor

“They were always open to answering my questions, no matter when they came! They gave me detailed explanations of anything I was confused about and never let me feel like I didn't know what I was doing. I was so thankful for their detailed feedback on my paper as I was writing it; my mentor would call attention to minor points and big ideas equally, and I think that my paper is the best that it can be because of them! They would always give me suggestions or tell me if I was on the right track if I was confused. My mentor was very flexible and was always open to working around my schedule.”