790046 Human gene therapy: Introduction (in Eng.)

Vorlesung und Seminar
Vortragende/r (Mitwirkende/r)
Hernandez-Bort, Juan-Antonio
Angeboten im Semester
Sommersemester 2022
Unterrichts-/ Lehrsprachen


Gene therapy is defined as the introduction of genetic material into the cells of patients to correct a genetically based defect or to endow them with a specific functionality. This emerging field is gaining increasing relevance in personalized medicine and brings additional challenges and opportunities for the academia and pharmaceutical companies. Currently, there is a gap for highly qualified scientists with wide knowledge in gene therapy. This course covers the most relevant aspects in the field to fill this gap.
-Basic concepts and modalities (viral / non-viral) in gene therapy
-Choosing the adequate vector for the treatment: Advantages and Disadvantages
-Expression systems for viral vector production
-Challenges in clinical manufacturing of Gene Therapy products
-Basics on formulation for gene therapy products
-Delivering the gene into the patient: Administration routes
-Clinical trials for gene therapy and ethical aspects
-Successes, failures and hopes
-Regulatory aspects to consider
-Recent history: Approved therapies
-The future of gene therapy

Inhaltliche Voraussetzungen (erwartete Kenntnisse)

Master and PhD students of biotechnology or related subjects (Bachelor students will not be accepted).
The course is recommended for students who already are familiar with molecular and cell biology concepts as well as biotechnological processes (see also “Teilnahmekriterien & Anmeldung”) and want to acquire a solid foundation in the field of human gene therapy.


Students acquire a solid academic foundation and competence in this field, being able to explain the different gene therapy modalities and understand the opportunities and limitations. They can rapidly identify the most relevant key performance indicators in a gene therapy biotechnological process and can provide input to improve them. Students have good understanding of potential analytical panels for the different modalities.
They know which are the most promising therapies and can describe how they can be applied to the patient. The graduates in this course not only know trusted information sources, such as journals and guidelines but also recognized societies in the field. They can explain the different clinical phases necessary to move towards gene therapy drug commercialization. Moreover, they have developed awareness about ethical aspects to be considered in the use of this emerging tool.
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