Alzheimer’s disease, Genome editing, Genetics, Genome SIIR News EP.2 Gene Therapy
This is iran in the national relations news from the success of the human genome project that was completed in 2003, there has been a major change in medical science. The knowledge allow the researcher to understand better about many diseases in terms of expression, severity and respond to the treatment among different population. Further, the unlocked information will pave the way to the new era of therapeutic modality, utilizing individual genetic background or what we call a physician medicine today is the greatest opportunity to have a chance to talk to one of the best experts in gene therapy and thalassemia. Professor dr vipra vipakasi professor of paediatric and program director for thalassemia and hemoglobin opacity research, so i declared professor v price. How are you today im very well? Thank you very much without any further ado. Let me start with the first question how thailand benefits from the human genome project, so the human genome project is one of the very important or scientific advancement in human history. Er in this program, we sequence around 3 billion bases in the human genome and that giving us the blueprint of the all the human fat diseases and all the phenotype. So, even though the program has finished since 2003, however, we just started to enjoy using the information from this program in order to improve our clinical practice in order to improve our diagnosis of many diseases and, of course, to use this information to improve our medical treatment.
To our patients and thailand is one of the country that we going to utilize this information to improve our medical care. Can you tell me the situation of the gene therapy in the international level comparing to thailand its a very, very important question? In the past 10 years, the gene therapy has increasingly important as one of the new treatment modalities for many diseases. So if you go into the international public data search, you will find that over 100 of the clinical gene therapy trial is now registered to the clinical uh government websites at the usa and at the moment this year, 25 uh gene therapy product has been registered to Get approval by the health authority, including the us fda and the ema for european countries, so the gene therapy has become you know one of a very fascinating and also very important new kind of treatment at the international level for thailand. We just started to be part of this. You know exciting holy grail for the new treatment modalities. Weve been part of are participating in several clinical trials, using gene therapy like last year that have already been published in the new england journal, medicine and also this year. At the rerun hospital, we also are doing some kind of gene therapy treatment for patients with thalassemia as well. Okay, i see next question what kind of diseases that can be treated with gene therapy, sir, actually, or to treat patients with gene therapy.
You might think that you know one of a very priority will be the disease which is caused by the genetic mutation. So, for this regard, our patients with mental genetic or the single gene disorder, such as thalassemia hemophilia, are spinal muscular atrophy and other single gene disorder. As a first priority to be treated with gene therapies and of course indeed, there are several programs that are aiming to treat patients with such a condition. However, technology has evolved. I have to say now: gene therapy has become more applicable for even more complex diseases as well, because we understand more on the gene regulation and we can manipulating the gene uh in a more robust and more efficiently. So in this regard, so there are so several programs to use gene therapy to treat neurodegenerative diseases, parkinsons disease, alzheimers or even more importantly, the cancer. So all of these conditions now are in the process to develop gene therapy to treat them all incredible next question: what is your ongoing research and how they are going to benefit iran and thailand? So at the moment i am running a group to establish an infrastructure for gene therapy for the right hospital. In order to do that, it is very important that we have to develop at the three different important aspects. So the first aspect is to develop the gene delivery system. So in our hospital we plan to use the adrenal viral vector and the transport sun vector our transport zone, vector which known as the sleeping beauty as the system to deliver the gene into the genome.
So thats the first part. The second part is how to monitor the gene that we did or we are adding into the genome. So in order to do that, we need to use the technology that we call next generation sequencing so were going to develop. The discovery. Seek in order to use and monitor the genome after the process of the gene therapy, because this is a very important in term of the safety when we manipulating the genome. The third part, also very important, is also what we are doing to develop. The clinical gene therapy, which is related to the transplantation process and as a part of that we are running at least two clinical trials with the international agency, especially related to the genome. Editing program called the crispr cas9 technology in patients with thalassemia and also the genome. Editing program by manipulating some of the iron regulating genes in order to improve the clinical severity in the patient. So these three important pillars are important and critical to establish the gene therapy program at civil rights hospital, and this is what i am doing currently. Okay last but not least, question uh. What do you expect to see or to happen in the future about the gene therapy at sea rat hospital? So it is very important and also a fundamental concept of what we are working here at the rewrite hospital that you know for what we develop in term. Of the technology, its not just the sake of the technology itself or just the publication that come out of that, but we need to be able to make such a technology become accessible and possible for most of the people here in this country.
So this is the concept that you know its, not just the process of the learning or the process of the technology, but its into the application to the benefit of the mankind. So we do hope that, with our program, we shall be able to make the gene therapy become accessible for most of the people in this country. Well, thank you very much, professor vipra, for your valuable knowledge today, and that is a latest update of the craft performance in order to make a difference to the people.