Gene Therapy for Thalassemia
Greece (HelMSIC) - National and Kapodistrian University of Athens, Athens
Laboratory of Biology, Medical School of Athens, University of Athens
Prof. E. Marinos
Eleni Papanikolaou
English, Italian
4 weeks
Cities/Months Jan Feb Mar Apr May Jun Jul Augt Sep Oct Nov Dec
No No Yes No No Yes No No No No No No
Type of Research Project
- Basic science
What is the background of the project?
Gene therapy refers to the use of DNA to treat a disease. In β-thalassemia the genes encoding the β-chains of hemoglobin are absent or non functional. This results in life threatening anemia that can be lethal if left untreated. In gene therapy protocols, the therapeutic gene, i.e. the β-globin gene in the case of β-thalassemia, is packaged within a vector that is used to facilitate its entrance in the patients
What is the aim of the project?
What techniques and methods are used?
What is the role of the student?
What are the tasks expected to be accomplished by the student?
Will there be any theoretical teaching provided (preliminary readings, lectures, courses, seminars etc)
What is expected from the student at the end of the research exchange? What will be the general outcome of the student?
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What skills are required of the student? Is there any special knowledge or a certain level of studies needed?
The students must have satisfactory knowledge in Hematology, Molecular Biology, Virology. Idially to have completed preclinical years. There are no legal limitatons in the student
Are there any legal limitations in the student’s involvement

Type of students accepted
This project accepts:
- Medical students
- Towards more successful gene therapy clinical trials for β-thalassemia. Drakopoulou E. Papanikolaou E . Georgomanoli M. Anagnou NP. Curr Mol Med. 2013 Sep;13(8):1314-30. Review.
- The new self-inactivating lentiviral vector for thalassemia gene therapy combining two HPFH activating elements corrects human thalassemic hematopoietic stem cells. Papanikolaou E. Georgomanoli M. Stamateris E. Panetsos F. Karagiorga M.