The promise and potential challenges of gene therapy
What role is gene remedy more likely to play in the future? We talk with Mentor Wolfgang Uckert, the chief executive director of the German Culture Contemporary society for Gene Therapy, about the potential of the treatment.
BLOG: The thought of gene remedy has been around for many years, but has seen only limited success. Why?
Wolfgang Uckert: I actually feel that one of the problems has been the transfer vector, which can be used to bring a new gene into a cellular. So right from the start, these vectors were not really safe.
BLOG:What is the risk associated with that?
The risk is that they assimilate in our genome at an unacceptable place and then we certainly have side effects and the medial side results can be other diseases, like leukemia for example.
That's a pretty heavy side effect.
BLOG:What can be done to reduce the potential risks or the aspect effects?
Yeah this already started years back - to increase the vectors. Now we have a new technology of vectors which are really safe because they can integrate in the certain point of the genome at specific sequences and we have basic safety modules that are included in the vectors and the genetically modified skin cells and that we can simply change off the vector and can eliminate the cellular.
BLOG:Is that then leading to greater approval of gene therapy?
Yes, We feel that it will be better gene remedy very much.
BLOG:Let's speak about some of the ethical issues encircling gene therapy. What are they?
The ethical issues are that one needs to select very well the disease, what type he/she wants to treat because this is a three-component system. You will need to manipulate a cell, you have to transport a gene into the cellular, and you have to generate a vector choice and that must be well-designed.
BLOG:Where will gene remedy look the most promising?In healing which diseases specifically?
And so we certainly have seen this season: these monogenetic diseases, so this means that only one gene must be corrected. This is not at all hard I would say: it's more difficult if we go to tumor, because cancer is a multi-factorial disease and many, many genes are included - and you have to cure each specific defect or gene.
What role is gene remedy more likely to play in the future? We talk with Mentor Wolfgang Uckert, the chief executive director of the German Culture Contemporary society for Gene Therapy, about the potential of the treatment.
BLOG: The thought of gene remedy has been around for many years, but has seen only limited success. Why?
Wolfgang Uckert: I actually feel that one of the problems has been the transfer vector, which can be used to bring a new gene into a cellular. So right from the start, these vectors were not really safe.
BLOG:What is the risk associated with that?
The risk is that they assimilate in our genome at an unacceptable place and then we certainly have side effects and the medial side results can be other diseases, like leukemia for example.
That's a pretty heavy side effect.
BLOG:What can be done to reduce the potential risks or the aspect effects?
Yeah this already started years back - to increase the vectors. Now we have a new technology of vectors which are really safe because they can integrate in the certain point of the genome at specific sequences and we have basic safety modules that are included in the vectors and the genetically modified skin cells and that we can simply change off the vector and can eliminate the cellular.
BLOG:Is that then leading to greater approval of gene therapy?
Yes, We feel that it will be better gene remedy very much.
BLOG:Let's speak about some of the ethical issues encircling gene therapy. What are they?
The ethical issues are that one needs to select very well the disease, what type he/she wants to treat because this is a three-component system. You will need to manipulate a cell, you have to transport a gene into the cellular, and you have to generate a vector choice and that must be well-designed.
BLOG:Where will gene remedy look the most promising?In healing which diseases specifically?
And so we certainly have seen this season: these monogenetic diseases, so this means that only one gene must be corrected. This is not at all hard I would say: it's more difficult if we go to tumor, because cancer is a multi-factorial disease and many, many genes are included - and you have to cure each specific defect or gene.
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