What to Expect
In recent years, medical science has made tremendous progress, particularly in the field of gene therapy. Particularly fascinating is the so-called “N-of-1” or “N=1” therapy, which is tailored to the genetic profile of one single person. This approach offers a new hope for children and adolescents with rare, previously incurable diseases such as PCH2.
Just recently, a child in the US with a rare genetic metabolic disorder was cured through personalized gene therapy, as reported in articles in The New York Times and CNN. Scientists around the world are working to develop personalized treatments that are tailored to the specific genetic characteristics of a single child. Their work focuses not only on making medical progress, but also on ethical issues and the importance of close cooperation between parents, doctors, and researchers.
Our online consultation hour “Meet the expert” gives you the opportunity to ask your questions about individualized gene therapy and its possible application in PCH2 during a live online meeting.
In our English online consultation on 24 June 2025 at 8 pm CET, Prof. Dr. Simone Mayer and Dr. Julia Matilainen will present recent findings on individualized gene therapy with N-of-1 and its potential application in PCH2. Afterwards, the speakers and their team will be available to answer your questions live in an open Q&A session.
Prof. Dr. Simone Mayer is a professor at the Karlsruhe Institute of Technology (KIT) and head of the Department of Systemic Cellular Neurobiology. Her research group studies the disease mechanisms of PCH2 using cerebellar organoids.
Dr. Julia Matilainen has a child with PCH2 and acts as Chief Scientific Officer for the PCH-Familie e.V. association. Her primary goal is to find treatment options for children with PCH2 in the near future. She has a PhD in Pharmaceutical Technology and works in the pharmaceutical industry in drug development, specifically in the field of cell and gene therapies.
To help us plan, please register for the event using the registration form below. You are welcome to send us your questions in advance using the “Comments” field in the registration form so that our experts can best prepare for the Q&A session.
We look forward to your participation!
Speakers
Prof. Dr.Simone Mayer
Dr. Julia Matilainen
Interested? Then register for free!
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