Genetic Therapy Approaches

With this course, you will learn about the current gene therapy technologies and the approaches you can use for the rare disease you are targeting. Gene augmentation therapy, CRISPR gene editing, suicide gene (suicide gene), human artificial chromosome, and anti-sense oligonucleotide (ASO) based approaches will be discussed among genetic treatment approaches. At the same time, approaches used within the scope of currently approved genetic therapies, Luxturna (Retinitis Pigmentosa), Zolgensma and Spinraza (SMA), and other treatments will be examined in detail.

Finally, we will discuss the difficulties encountered in genetic therapy approaches and what you should pay attention to before your genetic therapy projects. i. Gene Therapy: Introduction and Methodsii.

Gene Therapy Strategiesiii. Gene Therapy Methodsiv. Gene Targeting and Genetic Designv.

Gene Therapy in the Treatment of Diseasesvi. Challenges in Gene TherapyRadiChal's vision,"considered incurable">65 million people in the world with a genetic diseaseone-time recoveryTo provide a «one-shot cure»starting with the training of qualified specialists; to develop value-added products. Although there are approximately seven thousand rare diseases diagnosed; only less than 5% of these are thought to be curable.