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Prof. Dr. Ulrich Siler

Prof. Dr. Ulrich Siler

Prof. Dr. Ulrich Siler

Tätigkeiten an der FHNW

  • Dozent


  • Master: Gene and Cell Therapeutic Systems


In vivo Gene Delivery
Gene therapy represents a treatment option which allows to correct/compensate for the primary cause of a genetic disease. First clinical success in gene therapy was published in the year 2000. Since then, several gene therapy products such as Imlygic, Strimvelis, Kymriah, Luxturna and Zolgensma have received marketing approval, and others are about to be launched.
In ex vivo gene therapy, target cells are isolated from patient samples, genetically modified under GMP conditions, and then re-transplanted into the patient. In in vivo gene therapy, which targets the eye, brain or muscles for example, infectious particles are injected directly into the patient.
In ex vivo gene therapy, lentiviral vectors enveloped by vesicular stomatitis virus glycoprotein (VSVG) are state-of-the art and are applied ex vivo to target cells with proliferative potential (hematopoietic stem cells, T cells). While ex vivo gene therapy has established proof of principle, it faces an economic challenge: In addition to large-scale GMP-compliant vector production, personalized GMP-compliant ex vivo processing of patient cell increases the overall costs of treatment.

In vivo delivery would significantly reduce the total cost of treatment per patient.


Institut für Pharma Technology

Fachhochschule Nordwestschweiz FHNW Hochschule für Life Sciences Institut für Pharma Technology Hofackerstrasse 30 4132 Muttenz
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