Spinal Muscular Atrophy


Proje Name:

Treatment of Spinal Muscular Atrofi Disease with CRISPR Technology


The biggest development in the biological sciences is undoubtedly the discovery of the DNA double helix. This discovery in 1953 laid the groundwork for the study of structural changes in DNA, and with the development of technology, perhaps this structure will be a beacon of hope for most cancer diseases and rare diseases for humanity to quickly unravel in periods of progress. Over the last 20 years, various methods for DNA and RNA synthesis have been developed, and these methods have laid the groundwork for the discovery of genome organization. Polymerases, ligases, restriction enzymes, helicases and polymerase chain reaction (PCR) with the discovery of structures such as the isolation of genes and gene fragments, cell culture, model organisms and in vitro studies of gene mutations and gene maps are of great importance. With the advent of genomic sequencing, genome maps of humans and other organisms began to form. The Clustered Regularly Interspaced Palindromic repeats (CRISPR)-Cas system, a genome sequencing tool based on the principles of the Watson – Crick DNA model, has been of considerable importance to biological science. The CRISPR-Cas system has become a beacon of hope for humanity, especially for the treatment of rare diseases. In our study, it was aimed to repair the tissue associated with cutting the faulty area with the enzyme Nhae1 restriction over exon 7 using the CRISPR-Cas9 system for Spinal muscular atrophy disease and adding a properly prepared primer to the cut area. This mechanism is intended to minimize the toxicological effect of herpes viruses and proteins in the preparation and transfer into the cell.

Team Members

Azize ÖZEN

I graduated from Bartın University Molecular Biology and Genetics from 2019. Gaziosmanpaşa Universty Medical Biology Deparment. I try to improve myself by working for science and humanity, taking trainings and doing internships. In my future life, I will contiune with patience and devotion to serve both myself, my country and humanity by interwining with science.


I completed my bachelor degree on Molecular Biology and Genetics at Kilis 7 Aralık University, in 2019. I am currently master student in the department of Biotechnology at University of Çukurova. My interests are Molecular Genetics, Microbial Biotechnology, Epigenetics and Cancer Genetics.

Tutku Melis AYGÜN

I got Bachelor’s Degree from Molecular Biology and Genetics (English) of the Faculty of Engineering of Biruni University. As part of my undergraduate education, I worked as a volunteer, an intern and undergraduate researcher in many fields related to Molecular Genetics, Andrology and Embryology. My Master’s Degree continues in the Clinical Embryology program at İstanbul Yeni Yüzyıl University. Since my background focuses on the Molecular Genetics, Reproductive Genetics, Infertility, Andrology and Embryology, I aim to improve myself in these fields and broaden my perspectives in that direction. Also, I have a review article published in an international journal.

Şeyda Nur ŞAN

In the year of 2018, I completed my bachelor degree on Molecular Biology and Genetics at Bilecik Seyh Edebali University. At the university, I  focused on exploring incurable diseases such as cancer, facioscapulohumeral muscular dystrophy (FSHD), multiple sklerozis (MS) and the treatment methods. With my final thesis which was funded by TUBITAK, I explored the world of miRNAs which have important roles in gene regulation and I combined certain natural drugs on breast cancer cell lines in order to evaluate efficacy of drugs. I have experience several laboratories such as Physiology laboratory in Italy, Prostate Canser Laboratory in Finland, Biotechnology Laboratory and Cancer Laboratory inTurkey. I am currently studying Master degree in the department of Molecular and Cellular Biology at Sorbonne University.


I am senior undergrduate student at Uşak Universty, Faculty of Science and Letters Deparment of Molecular Biology and Genetics. While continuing my undergraduate education, I have been a volunteer internship in neurology, oncology ans stem cel related fields. My interests continue on these areas