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 curestarting 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. For this reason, it is indisputable how high the need for gene therapy methods is for individuals with rare diseases caused by genetic disorders that reduce their quality of life and shorten their lifespan. gene therapy; is accepted as a technique that changes a part of a person’s genetics to repair a defective gene in the genome of a person suffering from hereditary/familial rare diseases or to maintain their functional characteristics. With the revelation of the healthy human genome code at the beginning of the 2000s, the genetic disorders underlying many diseases came to light and the development of gene therapy methods to repair these damaged DNA sequences gained momentum. The gene therapy method, which can be defined as ’transferring synthetic gene copies that will replace mutated genes and fulfill their natural function to the target cells of the patient with viral or non-viral transfer methods’, is essential in achieving single-shot therapy compared to drugs or alternative medicine treatments. arguably has an advantage. For this reason, pharmaceutical and biotechnology companies have focused their treatment research in recent years, especially on gene therapy.
