Friedreich Ataxia

Prize: Silver Medal

Project Name:

Obtaining physiological frataxin expression in FRDA Fibroblast Cell Line with 2 alternative strategies: HSV-1/AAV Hybrid Amplicon Vector and Hybrid AAV Dual Vectors


In this project, we aimed at regulating frataxin (FXN) expression level in FRDA fibroblast cell line with HSV-1/AAV hybrid vector which contains mutant AAV2 rep and has advantageous due to large transgene capacity. Also with providing nontoxic and stable gene delivery, it will be more safe to use HSV-1/AAV hybrid vector for CNS-directed gene delivery.
In addition, we suggest that using FXN promoter and fragment of the FXN intron 1 on our design will be beneficial to obtain physiological frataxin expression. Also as a second strategy, AAV9 vectors are designed with hybrid dual vector approach which may have a potential to rescue frataxin deficiency with delivering large transgene and according to our observations, differs from the other AAV vector studies related with Friedreich’s Ataxia.
With analyzing advantageous and disadvantageous parts of both strategies, we hope these studies will be more applicable with further studies and be beneficial for potential gene therapy studies of FA.

Team Members

sude güneş

Sude Güneş

Born in 2000, Sude Güneş graduated from Private Ege High School in 2018. She is an undergraduate student at İzmir Institute of Technology (IZTECH) Bioengineering Department and will continue her undergraduate education as a third-year student in the 2020-2021 academic year. IZTECH
Bioengineering Student Community, Radio Community and Entrepreneurship Society of IZTECH are student communities that she participated.

ıtır güldağ

Itır Güldağ

Born in November 2000, Itır Güldağ graduated from Private Oğuzhan Özkaya Science High School with the second place in 2018. She is currently a third year student at Izmir Institute of Technology
(IZTECH) Bioengineering Department. While taking part in IZTECH Bioengineering Student Club and IZTECH Cinema Club she also actively assisted studies for a post graduate and PhD thesis conducted by Bulmuş Lab in IZTECH Bioengineering Department for about one month in the summer of 2020.

alara kara

Alara Kara

Born in 1999, Alara Kara graduated from 60th. Year Anatolian High School in 2018. In the 20202021 academic year, she will continue her undergraduate education as a 3rd grade at Izmir Institute of
Technology (IZTECH) Bioengineering Department.She plays an active role in the IZTECH musical society and folk dance society and takes part in performances. She is the vice president of the IZTECH
Bioengineering student society. She did an internship in the Özçivici Lab, while she was in second grade. She enjoys taking part in Bioengineering and Genetics projects.