METAGEN

Glycogen Storage Disease

Prize: Best Social Awareness

Silver Medal

Project Name:

METALINE: Gene Therapy Applied to Differentiated Hepatocyte Cells

Abstract

Glycogen storage disease (GSD) type 1a is an autosomal recessively inherited genetic disease caused by glucose-6-phosphatase enzyme deficiency. There is no treatment for GDH 1a. Current treatment is a diet fortified with alternative medications. Corn starch nutrition is given at 6 months to prevent hypoglycemia and increase blood sugar. A gene therapy that differentiates into hepatocytes with stem cells in glycogen storage disease type 1 has not yet been performed. Mesenchymal stem cells are obtained from adipose tissue taken from individuals with glycogen storage disease type 1a. Adipose tissue is preferred because adipose tissue has more mesenchymal stem cells than bone marrow, has genetic stability, is easy to obtain, mesenchymal stem cells obtained from adipose tissue are non-immunogenic in the treatment of many diseases and in regeneration of damaged tissue. Epidermal growth factor (EGF), fibroblast growth factor (FGF), nicotinamide (NTA), insulin transferin selenium (ITS), dexamethasone (DEXA) and hepatocyte growth factor (HGF) are used to differentiate mesenchymal stem cells into hepatocyte cells in vitro. Gene augmentation therapy is used as gene therapy. GFP gene is used in vitro to see in mesenchymal and hepatocyte cells of our gene augmentation therapy. It is transferred to our hepatocyte and mesenchymal stem cells via lentivirus. 4th Generation (self inactivion) ​​plasmids are used to reduce the risk of oncogenesis of lentivirus. Therefore, our lentivirus can be self-inactivated by deleting a few bases in the 5’LTR and 3’LTR regions. Instead of repairing the intensive mutation with gene enhancement therapy, it can repair all mutations in exons in the G6PC gene. It is shown in molecular genetic testing and mouse experiments that our cellular genetic therapy works. In mouse experiments, the luciferase enzyme is used in vivo instead of the GFP gene. Complications such as hypoglycemia and hepatomegaly in mice are tested and evaluated according to nutritional status.

Team Members

besne çelik

BESNE ÇELİK

AFYON KOCATEPE UNIVERSITY
FACULTY OF SCIENCE AND LITERATURE
MOLECULAR BIOLOGY AND GENETICS DEPARTMENT 4TH GRADE

buket güzel

BUKET GÜZEL 

MARMARA UNIVERSITY
FACULTY OF SCIENCE AND LITERATURE
BIOLOGY DEPARTMENT 3TH GRADE

buket uysal

BUKET UYSAL

HITIT UNIVERSITY
FACULTY OF SCIENCE AND LITERATURE
MOLECULAR BIOLOGY AND GENETICS 4TH GRADE

huriye akın

HURİYE AKIN

BARTIN UNIVERSITY
FACULTY OF SCIENCE
MOLECULAR BIOLOGY AND GENETICS 3TH GRADE

büşra can

BÜŞRA CAN

RECEP TAYYIP ERDOĞAN UNIVERSITY
FACULTY OF MEDICINE, 3TH GRADE