Glycogen Storage Disease

Prize: Silver Medal

Project Name:

Primum Non Nocere!


GSD Type1A occurs when the G6PC enzyme can’t function properly as a result of over 100mutations in the G6PC gene. Instead of focusing on these mutations individually, we‘ll transfer the functional G6PC gene to the mutated cells to create a treatment. Alternatively, we’ll transfer a more active isoform of G6PC(S298C) and use the more effective one in our project by comparing two of them. We chose lentiviral vectors with high carrying capacity to transfer the gene at once. We used integrase defective 3rd generation lentiviral vectors to avoid oncogenic effects of lentiviral vectors. To avoid decreasing of effectiveness of the episomal genetic treatment over time, we’ll integrate the G6PC gene into the genome using the CRISPR/dCas9-FokI, which has the least risk of off-target mutations. We plan to put the G6PC gene into the G6PC gene region or the safe harbor region AAVS1. We decided the intravenous(IV) route is more practical and effective for in-vivo gene therapy. However, since the intravenous route isn’t local, our vector may spread different tissues and G6PC synthesize in unwanted tissues and cause metabolic disorders. Therefore, we designed the envelope proteins of lentiviral vectors, and added miRNA target regions, so that our treatment will be specific to the liver. In addition, we used tetracycline dependent liver specific promoters to minimize the potential risks of continuous systems. By adhering to the ‘Primum Non Nocere’ principle, we made designs to increase the safety of our project at every stage, and planned to treat GSD Type1A ideally.

Team Members

ömer enes önür

Ömer Enes ÖNÜR

My name is Ömer Enes, I live in Bursa. I graduated from Uludağ University Faculty of Medicine. I am currently working as a general practitioner in the emergency room at Bursa Yenişehir State Hospital. I am very interested in music and sports.

burcu nur başdan

Burcu Nur BAŞDAN

My name is Burcu, I live in Ankara. I am a 5th grade student at Hacettepe University Faculty of Medicine. I like to play table tenis and play a musical instrument. I also like to watch TV series in my spare time. And my goal is to be a good doctor.

melike demir

Melike DEMİR

My name is Melike, I live in Erzurum. I am a 5th grade student at Eskişehir Osmangazi University Faculty of Medicine. I am interested in molecular biology and genetics. I want to be a pediatrician.

abdullah demir

Abdullah DEMİR

My name is Abdullah, I was born on November 8, 1997 in Tokat. I live in Eskişehir. I am a 6th grade student at Eskişehir Osmangazi University Faculty of Medicine. I’m interested in chess. I like watching movies and playing basketball. I love to play games with my friends in my spare time. I want to be a good person and a good doctor.

furkan sait ceylan

Furkan Sacit CEYLAN

Hello 🙂 I am Furkan Sacit Ceylan. My last year at Hacettepe University Faculty of Medicine. I am interested in molecular biology and microbiology. In the future, I plan to become a clinical microbiology and infection specialist and to produce new treatments in this field with molecular biology.