Personalized Gene Therapy

The ability to switch disease-causing genes on and off remains a dream for many physicians, research scientists and patients – which may become a reality sooner than anticipated.

Research teams from across the world are busy turning this dream into a reality, including a team of researchers from Charité — Universitätsmedizin Berlin and the Max Planck Institute for Medical Research in Heidelberg. The team has successfully programmed a virus to transport the necessary genetic material to affected tissue and nerve cells inside the body.

A report on their new virus-based method, which delivers instructions to the host genome without becoming part of it, has been published in the journal Molecular Therapy Nucleic Acids.

From cancer to Alzheimer’s disease, many life-threatening diseases can only be treated using drug-based treatment options, if at all. Many of these treatments are non-specific in nature, or even ineffective. In some cases, the undesirable side-effects may even outweigh the desirable ones. This is because indiscriminate treatments damage healthy cells, impairing their ability to communicate with other cells; as a result, it is hoped that genetically produced and modified mediators will be able to selectively target diseased cells, and improve the way treatment is delivered. Using attenuated, i.e. non-replicating, viruses that are known as recombinant adeno-associated viruses (rAAV) – allows for the transport of genetically encoded material into live organisms affected by disease. This approach opens up a whole range of options which, in the future, may allow for the treatment of various chronic diseases.

By successfully completing the initial step of testing this new method using an animal model, the researchers have laid the groundwork for future genetic treatments for use in humans. Before these can be used, however, they will need to be tested to ensure their safety. It is already known that rAAVs can transport genetically encoded material into any type of cell and tissue, including the brain, and that, once inside the cells, they are capable of repeatedly switching gene therapy applications on and off again. This on/off switch is controlled chemically, via either food intake or drinking water. The gene function can be switched on and off in this manner, which is of particular value, and renders the method a perfect candidate for use in controlled gene therapy.

The fact that rAAV-infected cells do not trigger any form of measurable immune response and that their genetic material remains completely intact represents an additional benefit. While this does not mean that future gene therapy applications are guaranteed to be successful, the researchers are full of confidence for the future. While still at the laboratory stage, this development could spearhead innovation, heralding in a time when the transfer of genetically encoded material will be used to heal severe diseases, including neurological ones, such as Parkinson’s disease, Alzheimer’s disease and epilepsy.

For more information on personalized gene therapy, join me at Cell & Gene Therapy Bioprocessing & Commercialization this October in Boston. And for special savings, be sure to use the VIP code “B16188BLOG” – See you this October.



About the author: Justin Gaines is a Boston-based drug discovery professional and biotech enthusiast. During his tenures he has remained active within the biomedical engineering and regenerative medicine sectors, and has remained an avid proponent of bringing together the Boston life science community. He is currently leading the Bio Pharma Networking Group across the North East – a professional life science and healthcare community spanning 6K members. View his last contribution to KNect365 Bioprocessing here



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